The information in this preliminary proxy statement/prospectus is not complete and may be changed. Neoleukin may not sell the securities described in this preliminary proxy statement/prospectus until the registration statement filed with the Securities and Exchange Commission is effective. This preliminary proxy statement/prospectus is not an offer to sell and it is not soliciting an offer to buy these securities in any jurisdiction where the offer or sale is not permitted.

PRELIMINARY PROXY STATEMENT/PROSPECTUS

PROPOSED MERGER

YOUR VOTE IS VERY IMPORTANT

To the Stockholders of Neoleukin Therapeutics, Inc. and Neurogene Inc.,

Neoleukin Therapeutics, Inc., a Delaware corporation (“Neoleukin”), Project North Merger Sub, Inc., a Delaware corporation and wholly owned subsidiary of Neoleukin (“Merger Sub”), and Neurogene Inc., a Delaware corporation (“Neurogene”), entered into an Agreement and Plan of Merger (the “Merger Agreement”) on July 17, 2023, pursuant to which, among other matters, and subject to the satisfaction or waiver of the conditions set forth in the Merger Agreement, Merger Sub will merge with and into Neurogene, with Neurogene surviving as a wholly owned subsidiary of Neoleukin (such transaction, the “merger”). After the completion of the merger, Neoleukin will change its corporate name to “Neurogene Inc.” The surviving company following the merger is referred to herein as the “combined company.”

At the effective time, upon the terms and subject to the conditions set forth in the Merger Agreement, each warrant to purchase Neurogene common stock (“Neurogene pre-funded warrant”) will be automatically converted into and exchanged for a Neoleukin pre-funded warrant entitling such holder of the Neurogene pre-funded warrant to purchase shares of Neoleukin common stock equal to the product of the number of shares of Neurogene common stock that would have been issuable upon exercise of the Neurogene pre-funded warrant and the exchange ratio, and such Neoleukin pre-funded warrant shall have an exercise price equal to the exercise price per share of the Neurogene pre-funded warrant divided by the exchange ratio, as described in more detail in the sections entitled “The Merger Agreement—Merger Consideration” and “The Merger Agreement—Exchange Ratio” beginning on page 156 of this proxy statement/prospectus.

The final exchange ratio is subject to adjustment prior to closing of the merger (the “closing”) based upon Neoleukin’s net cash, as defined in the Merger Agreement (“Neoleukin’s net cash”) at closing as well as the aggregate proceeds from the sale of Neurogene common stock and pre-funded warrants in the Neurogene pre-closing financing and, as a result, Neoleukin securityholders could own more, and Neurogene securityholders (including, for this purpose, investors in the Neurogene pre-closing financing) could own less, or vice versa, of the combined company. Based on Neoleukin’s and Neurogene’s capitalization as of July 17, 2023, the date the Merger Agreement was executed, after giving effect to a 1-for-5 reverse stock split for Neoleukin common stock

that was effected on September 25, 2023, the exchange ratio was estimated to be equal to approximately 0.3120x shares of Neoleukin common stock for each share of Neurogene capital stock, which estimated exchange ratio did not give effect to the expected Neoleukin reverse stock split that Neoleukin’s stockholders are being asked to approve pursuant to the proxy statement that is a part of this registration statement on Form S-4. Neoleukin management continues to anticipate that Neoleukin’s net cash at the closing will be not less than the Target Parent Net Cash (as defined in the Merger Agreement) amount and Neurogene’s management continues to anticipate that the aggregate proceeds from the pre-closing financing will be approximately $95.0 million, and, as further described below and in connection with the Merger Agreement, if both assumptions are accurate, there would be no adjustment to the exchange ratio.

In connection with the merger, Neoleukin will assume Neurogene’s 2018 Equity Incentive Plan (“2018 Plan”). Each outstanding and unexercised option to purchase shares of Neurogene common stock immediately prior to the effective time will be assumed by Neoleukin and will be converted into an option to purchase shares of Neoleukin’s common stock, with necessary adjustments to the number of shares and exercise price to reflect the exchange ratio.

In connection with the merger, each share of Neoleukin common stock that is issued and outstanding at the effective time will remain issued and outstanding. Each unexpired, unexercised and unvested option to purchase Neoleukin common stock (“Neoleukin option”) that has an exercise price per share less than $18.90 (after giving effect to the 1-for-5 reverse stock split of Neoleukin common stock that was effected on September 25, 2023) that is held by a current employee, director or consultant of Neoleukin as of immediately prior to the effective time or who ceases to be a current employee, director or consultant of Neoleukin as of immediately prior to the effective time, will be accelerated in full as of immediately prior to the effective time. All outstanding Neoleukin restricted stock units (“RSUs”) that vest solely on the basis of time that are outstanding immediately prior to the effective time will be accelerated in full and will be settled in shares of Neoleukin common stock immediately prior to the effective time, contingent upon the occurrence of the closing.

Immediately after the merger, Neoleukin securityholders as of immediately prior to the merger are expected to own approximately 16% of the outstanding shares of capital stock of the combined company and Neurogene securityholders as of immediately prior to the merger are expected to own approximately 84% of the outstanding capital stock of the combined company, with former Neurogene securityholders, excluding shares of Neurogene common stock and Neurogene pre-funded warrants purchased in the Neurogene pre-closing financing, expected to own approximately 57% of the total outstanding shares of capital stock of the combined company and shares of Neurogene common stock and Neurogene pre-funded warrants issued in the Neurogene pre-closing financing expected to represent approximately 27% of the outstanding shares of capital stock of the combined company, subject to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash amount and the aggregate proceeds of the Neurogene pre-closing financing being $95.0 million. The foregoing percentages are calculated using the Treasury Stock Method (“TSM”).

Shares of Neoleukin common stock are currently listed on The Nasdaq Capital Market (“Nasdaq”) under the symbol “NLTX.” Neoleukin intends to file an initial listing application for the combined company with Nasdaq. After completion of the merger, Neoleukin will be renamed “Neurogene Inc.” and it is expected that the common

stock of the combined company will trade on Nasdaq under the symbol “NGNE.” On     , 2023, the last trading day before the date of the accompanying proxy statement/prospectus, the closing sale price of Neoleukin common stock was $  per share.

The closing of the Neurogene pre-closing financing is conditioned upon the satisfaction or waiver of the conditions to the closing of the merger as well as certain other conditions. The closing of the merger is conditioned upon the closing of the Neurogene pre-closing financing, with gross proceeds to Neurogene of at least $75.0 million, as well as certain other conditions. The Neurogene pre-closing financing is more fully described in the accompanying proxy statement/prospectus.

Neoleukin stockholders are cordially invited to attend the special meeting of Neoleukin stockholders. Neoleukin is holding its special meeting of stockholders (the “Neoleukin special meeting”) on    , 2023, at    Pacific Time, unless postponed or adjourned to a later date, in order to obtain the stockholder approvals necessary to complete the merger and related matters. The Neoleukin special meeting will be held entirely online. Neoleukin stockholders will be able to attend and participate in the Neoleukin special meeting online by visiting www.virtualshareholdermeeting.com/NLTX2023SM, where they will be able to listen to the meeting live, submit questions and vote. At the Neoleukin special meeting, Neoleukin will ask its stockholders to:

As described in the accompanying proxy statement/prospectus, certain Neoleukin stockholders who in the aggregate owned approximately 21% of the outstanding shares of capital stock of Neoleukin as of July 17, 2023, and certain Neurogene stockholders who in the aggregate owned approximately 77% of the outstanding shares of Neurogene capital stock as of July 17, 2023, are parties to stockholder support agreements with Neoleukin and Neurogene, respectively, whereby such stockholders have agreed to vote in favor of the adoption of the Merger Agreement and the approval of the merger and related transactions contemplated by the Merger Agreement, subject to the terms of the support agreements. Following the effectiveness of the registration statement on Form S-4 of which the accompanying proxy statement/prospectus is a part and pursuant to the Merger Agreement, Neurogene stockholders holding a sufficient number of shares of Neurogene capital stock to adopt the Merger Agreement and approve the merger and related transactions will be asked to execute written consents providing for such adoption and approval.

After careful consideration, each of the Neoleukin and Neurogene boards of directors have approved the Merger Agreement and have determined that it is advisable to consummate the merger. Neoleukin’s board of directors has approved the proposals described in the accompanying proxy statement/prospectus and unanimously recommends that its stockholders vote “FOR” the proposals described in the accompanying proxy statement/prospectus.

More information about Neoleukin, Neurogene, the Merger Agreement and transactions contemplated thereby and the foregoing proposals is contained in the accompanying proxy statement/prospectus. Neoleukin urges you to read the accompanying proxy statement/prospectus carefully and in its entirety. IN PARTICULAR, YOU SHOULD CAREFULLY CONSIDER THE MATTERS DISCUSSED UNDER “RISK FACTORS” BEGINNING ON PAGE 29 OF THE ACCOMPANYING PROXY STATEMENT/PROSPECTUS.

Neoleukin and Neurogene are excited about the opportunities the merger brings to Neoleukin’s and Neurogene’s stockholders and thank you for your consideration and continued support.

Neither the Securities and Exchange Commission nor any state securities commission has approved or disapproved of these securities or passed upon the adequacy or accuracy of the accompanying proxy statement/prospectus. Any representation to the contrary is a criminal offense.

The accompanying proxy statement/prospectus is dated    , 2023, and is first being mailed to Neoleukin’s stockholders on or about     , 2023.

NEOLEUKIN THERAPEUTICS, INC.

188 East Blaine Street, Suite 450

Seattle, Washington 98102

NOTICE OF SPECIAL MEETING OF STOCKHOLDERS

To the stockholders of Neoleukin Therapeutics, Inc.:

NOTICE IS HEREBY GIVEN that a virtual special meeting of stockholders (the “Neoleukin special meeting”) will be held on     , 2023 at    Pacific Time, unless postponed or adjourned to a later date. The Neoleukin special meeting will be held entirely online. You will be able to attend and participate in the Neoleukin special meeting online by visiting www.virtualshareholdermeeting.com/NLTX2023SM, where you will be able to listen to the meeting live, submit questions and vote.

The Neoleukin special meeting will be held for the following purposes:

Neoleukin’s board of directors has fixed     , 2023 as the record date for the determination of stockholders entitled to notice of, and to vote at, the Neoleukin special meeting and any adjournment or postponement thereof. Only holders of record of shares of Neoleukin common stock at the close of business on the record date are entitled to notice of, and to vote at, the Neoleukin special meeting. At the close of business on the record date, Neoleukin had    shares of common stock outstanding and entitled to vote.

Even if you plan to virtually attend the Neoleukin special meeting, Neoleukin requests that you sign and return the enclosed proxy or vote by mail or online to ensure that your shares will be represented at the Neoleukin special meeting if you are unable to virtually attend. You may change or revoke your proxy at any time before it is voted at the Neoleukin special meeting.

NEOLEUKIN’S BOARD OF DIRECTORS HAS UNANIMOUSLY DETERMINED AND BELIEVES THAT EACH OF THE PROPOSALS OUTLINED ABOVE IS FAIR TO, IN THE BEST INTERESTS OF, AND ADVISABLE TO NEOLEUKIN AND ITS STOCKHOLDERS AND HAS APPROVED EACH SUCH PROPOSAL. NEOLEUKIN’S BOARD OF DIRECTORS UNANIMOUSLY RECOMMENDS THAT NEOLEUKIN STOCKHOLDERS VOTE “FOR” EACH SUCH PROPOSAL.

The proxy statement/prospectus is available at www.virtualshareholdermeeting.com/NLTX2023SM.

By Order of Neoleukin’s Board of Directors,

Donna M. Cochener

Interim Chief Executive Officer, General Counsel

    , 2023

TABLE OF CONTENTS

QUESTIONS AND ANSWERS ABOUT THE MERGER

Except where specifically noted, the following information and all other information contained in this proxy statement/prospectus gives effect to the 1-for-5 reverse stock split approved by Neoleukin stockholders at the 2023 annual meeting which was effected on September 25, 2023, but does not give effect to the proposed reverse stock split described in Proposal No. 2 of this proxy statement/prospectus.

The following section provides answers to frequently asked questions about the merger. This section, however, provides only summary information. For a more complete response to these questions and for additional information, please refer to the cross-referenced sections.

In connection with the merger, Neoleukin will assume Neurogene’s 2018 Plan. Each outstanding and unexercised option to purchase shares of Neurogene common stock immediately prior to the effective time will be assumed by Neoleukin and will be converted into an option to purchase shares of Neoleukin’s common stock, with necessary adjustments to the number of shares and exercise price to reflect the exchange ratio.

In connection with the merger, each share of Neoleukin common stock that is issued and outstanding at the effective time will remain issued and outstanding. Each unexpired, unexercised and unvested Neoleukin option that has an exercise price per share less than $18.90 (after giving effect to the 1-for-5 reverse stock split of Neoleukin common stock that was effected on September 25, 2023) that is held by a current employee, director or consultant of Neoleukin as of immediately prior to the effective time, or who ceases to be a current employee, director or consultant of Neoleukin as of immediately prior to the effective time, will be accelerated in full as of immediately prior to the effective time, contingent upon the occurrence of the closing. All

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outstanding Neoleukin RSUs that vest solely on the basis of time that are outstanding immediately prior to the effective time will be accelerated in full and will be settled in shares of Neoleukin common stock immediately prior to the effective time, contingent upon the occurrence of the closing.

Immediately after the merger, Neoleukin securityholders as of immediately prior to the merger are expected to own approximately 16% of the outstanding shares of capital stock of the combined company and former Neurogene securityholders as of immediately prior to the merger are expected to own approximately 84% of the outstanding capital stock of the combined company, with former Neurogene securityholders, excluding shares of Neurogene common stock and Neurogene pre-funded warrants purchased in the Neurogene pre-closing financing, expected to own approximately 57% of the total of the outstanding shares of capital stock of the combined company and shares of Neurogene common stock and Neurogene pre-funded warrants issued in the Neurogene pre-closing financing expected to represent approximately 27% of the outstanding shares of capital stock of the combined company, subject in each case to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash amount and the aggregate proceeds from the Neurogene pre-closing financing being $95.0 million. The foregoing percentages were calculated using the TSM.

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Additionally, at least one of the following proposals must be approved to have an adequate number of authorized but unissued shares of Neoleukin common stock to complete the merger:

The issuance of Neoleukin common stock in connection with the merger and the change of control of Neoleukin resulting from the merger will not take place unless Proposal No. 1 and at least one of Proposal Nos. 2 or 4 are approved by Neoleukin stockholders and the merger is consummated. The amendment to the Neoleukin charter to effect a reverse stock split of Neoleukin’s issued and outstanding common stock will not take place unless Proposal No. 2 is approved by the requisite Neoleukin stockholders. The amendment to the Neoleukin charter to increase the number of authorized shares of Neoleukin common stock will not take place unless Proposal No. 4 is approved by the requisite Neoleukin stockholders. The Neoleukin board of directors may determine to effect the reverse stock split and/or the authorized share increase if they are approved and Proposal No. 1 is not approved by Neoleukin stockholders, following the special meeting.

The presence, by accessing online or being represented by proxy, at the Neoleukin special meeting of the holders of a majority of the shares of Neoleukin common stock outstanding and entitled to vote at the Neoleukin special meeting is necessary to constitute a quorum at the meeting for the Proposals.

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The presence, by accessing online or being represented by proxy, at the Neoleukin special meeting of the holders of a majority of the shares of Neoleukin common stock outstanding and entitled to vote at the Neoleukin special meeting is necessary to constitute a quorum at the meeting for the purpose of approving the Proposals.

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At or prior to the effective time, Neoleukin, a lease representative and a rights agent will enter into a Contingent Value Rights Agreement (the “CVR Agreement”), pursuant to which pre-merger Neoleukin common stockholders, holders of Neoleukin options outstanding as of the closing that are exercised after the closing, subject to the terms and conditions set forth in the CVR Agreement, and holders of Neoleukin existing pre-funded warrants outstanding as of the closing (“Neoleukin existing pre-funded warrants”) will receive one non-transferable CVR for each outstanding share of Neoleukin common stock held by such stockholder, option holder or Neoleukin warrant holder on such date. The contingent payments under the CVR Agreement, if they become payable, will become payable to the rights agent for subsequent distribution to the holders of the CVRs. In the event that no proceeds are received, or if not received within the periods specified in the CVR Agreement, holders of the CVRs will not receive any payment pursuant to the CVR Agreement. There can be no assurance that any holders of CVRs will receive payments with respect thereto.

The right to the contingent payments contemplated by the CVR Agreement is a contractual right only and will not be transferable, except in the limited circumstances specified in the CVR Agreement. The CVRs will not be evidenced by a certificate or any other instrument and will not be registered with the SEC. The CVRs will not have any voting or dividend rights and will not represent any equity or ownership interest in Neoleukin or the combined company or any of its affiliates. No interest will accrue on any amounts payable in respect of the CVRs.

For a more detailed description of the CVRs and the CVR Agreement, see “Agreements Related to the Merger—Contingent Value Rights Agreement” elsewhere in this proxy statement/prospectus.

Each share of Neoleukin common stock issued and outstanding at the time of the merger will remain issued and outstanding.

Neoleukin’s 2014 Equity Incentive Plan (the “Neoleukin 2014 Plan”) will continue on following the Merger Agreement, although assuming the 2023 Neurogene Equity Incentive Plan set forth in Proposal 5 is approved by the Neoleukin stockholders entitled to vote at the special meeting, no additional awards would be issued under the Neoleukin 2014 Plan after the effective time. Prior to the closing of the merger, and in connection with the merger, the Neoleukin board of directors will take actions to, contingent upon the occurrence of the closing, (i) accelerate the vesting of each then-outstanding unexpired, unexercised and unvested Neoleukin option that has an exercise price per share less than $18.90 (after giving effect to the 1-for-5 reverse stock split that was effected on September 25, 2023) that is held by a current employee, director or consultant of Neoleukin as of immediately prior to the effective time, or who ceases to be a current employee, director or consultant of Neoleukin as of immediately prior to the effective time, subject to the terms and conditions set forth in the Merger Agreement, (ii) accelerate the vesting of any unvested, time-based Neoleukin RSUs that are held by a current employee, director or consultant of Neoleukin as of immediately prior to the closing, or who ceases to be a current employee, director or consultant of Neoleukin as of immediately prior to the closing and (iii) deliver to the holders of such Neoleukin RSUs a number of shares of Neoleukin common stock equal to the number of vested and unsettled shares underlying such Neoleukin RSUs, in each case, in accordance with the terms of the Merger Agreement.

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In addition, under the Merger Agreement, no Neoleukin employee who is not already a participant in any offering period under the Neoleukin ESPP in effect as of the date of the Merger Agreement (the “Current ESPP Offering Period”) may become a participant and no participant may increase the amount of his or her payroll deduction election from that amount in effect on the date of the Merger Agreement for such Current ESPP Offering Period. The Neoleukin ESPP will be suspended and no new offering period will commence under the Neoleukin ESPP prior to the termination of the Merger Agreement. If any Current ESPP Offering Period is still in effect at the effective time, then the last day of such Current ESPP Offering Period will be accelerated to a date before the closing as determined by the Neoleukin board of directors (or relevant committee thereof) in its discretion.

Pursuant to the Merger Agreement, all Neoleukin existing pre-funded warrants that are outstanding immediately prior to the effective time will survive the closing and remain outstanding in accordance with their terms.

Neoleukin will assume Neurogene’s 2018 Plan. Neurogene option holders’ outstanding and unexercised options to purchase shares of Neurogene common stock immediately prior to the effective time will be assumed by Neoleukin and each outstanding and unexercised option will be converted into an option to purchase shares of Neoleukin’s common stock, with necessary adjustments to the number of shares and exercise price to reflect the exchange ratio.

Applying the exchange ratio, the former Neurogene securityholders immediately before the merger are expected to own approximately 84% of the aggregate number of shares of the combined company’s capital stock following the merger, with approximately 57% of the aggregate ownership allocated to securities of Neurogene outstanding prior to the pre-closing financing and approximately 27% of the total ownership allocated to shares of Neurogene common stock and Neurogene pre-funded warrants issued in the pre-closing financing, and Neoleukin securityholders immediately before the merger are expected to own approximately 16% of the aggregate number of shares of the combined company capital stock following the merger, in each case subject to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash amount and aggregate proceeds of $95.0 million from the Neurogene pre-closing financing. The foregoing percentages were calculated using the TSM.

For a more complete description of the treatment of Neurogene common stock, Neurogene options, Neurogene’s 2018 Plan and Neurogene pre-funded warrants in the merger, please see the sections entitled “The Merger Agreement—Merger Consideration” and “The Merger Agreement—Exchange Ratio”

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If you are a Neoleukin stockholder of record, you may provide your proxy instructions in one of four different ways:

Your signed proxy card, telephonic proxy instructions, or internet proxy instructions must be received by    , 2023 at 11:59 p.m. Eastern Time to be counted.

If you hold your shares in “street name” (as described below), you may provide your proxy instructions via telephone or the internet by following the instructions on your vote instruction form provided by your bank, broker or other nominee, referred to herein as “broker.” Please provide your proxy instructions only once, unless you are revoking a previously delivered proxy instruction, and as soon as possible so that your shares can be voted at the Neoleukin special meeting.

Whether or not you plan to attend the Neoleukin special meeting, we urge you to vote by proxy to ensure your vote is counted. You may still attend the Neoleukin special meeting even if you have already voted by proxy.

Please note that if you have more than one account through which you hold shares, you will receive more than one control number. The control number is used to vote your shares, and is also used to log on to the meeting website to virtually attend the meeting, which will allow you to vote the shares held in the account associated with that control number at the meeting. However, you will not be able to vote shares held in other accounts not associated with the control number you are using to log in to the virtual shareholder meeting. Therefore, it is important that you return your proxy cards for all of your accounts prior to the Neoleukin special meeting so that all of your shares may be counted.

If you are the registered stockholder and do not vote in one of the ways described above, your shares will not be voted at the Neoleukin special meeting and will not be counted toward the quorum requirement.

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Beneficial Owner: Shares Registered in the Name of a Broker, Fiduciary or Custodian

If you are the beneficial owner and do not direct your broker, fiduciary or custodian how to vote your shares, your broker, fiduciary or custodian will only be able to vote your shares with respect to proposals considered to be “routine.” Your broker, fiduciary or custodian is not entitled to vote your shares with respect to “non-routine” proposals, which we refer to as a “broker non-vote.” Whether a proposal is considered routine or non-routine is subject to stock exchange rules and final determination by the stock exchange. Even with respect to routine matters, some brokers are choosing not to exercise discretionary voting authority. As a result, we urge you to direct your broker, fiduciary or custodian how to vote your shares on all proposals to ensure that your vote is counted.

Please note that if you hold shares in more than one account, you will receive a different control number for each account. You may log in to the Neoleukin special meeting using any of your control numbers, however, you will only be able to vote the shares associated with that control number at the Neoleukin special meeting. Therefore, we encourage you to submit your votes in advance of the meeting for all accounts you hold to ensure your vote is counted at the Neoleukin special meeting. Please note if you do not provide any instructions to your broker to vote your shares on any proposal, your shares may not be voted at all and therefore would not be considered present at the meeting for the purposes of establishing a quorum to take action at the meeting.

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Your signed proxy card, telephonic proxy instructions, internet proxy instructions, or written notice must be received by    , 2023, 11:59 p.m. Eastern Time to be counted.

If a Neoleukin stockholder who owns Neoleukin shares in “street name” has instructed a broker to vote its shares of Neoleukin common stock, the stockholder must follow directions received from its broker to change those instructions.

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Neoleukin Therapeutics, Inc.

188 East Blaine Street, Suite 450

Seattle, Washington 98102

Telephone: (866) 245-0312

Attention: Corporate Secretary

Email: corporatesecretary@neoleukin.com

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RISK FACTORS

Risks Related to the Merger

The exchange ratio will not change or otherwise be adjusted based on the market price of Neoleukin common stock as the exchange ratio depends on the Neoleukin net cash at the closing and proceeds received by Neurogene Inc., a Delaware corporation (“Neurogene”) in the pre-closing financing, not the market price of Neoleukin common stock. Therefore, the merger consideration at the closing may have a greater or lesser value than at the time the Merger Agreement (as defined below) was signed.

On July 17, 2023, Neoleukin, Project North Merger Sub, Inc., a Delaware corporation and wholly owned subsidiary of Neoleukin (“Merger Sub”), and Neurogene entered into an Agreement and Plan of Merger (the “Merger Agreement”), pursuant to which Merger Sub will merge with and into Neurogene, with Neurogene surviving as a wholly owned subsidiary of Neoleukin. At the effective time, as described in the Merger Agreement, outstanding shares of Neurogene capital stock and prefunded warrants will be converted into shares of Neoleukin common stock or prefunded warrants to purchase Neoleukin common stock. Applying the exchange ratio, the former Neurogene securityholders immediately before the merger, including shares of Neurogene common stock and Neurogene pre-funded warrants to be issued in the Neurogene pre-closing financing, are expected to own approximately 84% of the aggregate number of shares of Neoleukin capital stock, and Neoleukin securityholders immediately before the merger are expected to own approximately 16% of the aggregate number of shares of Neoleukin capital stock calculated using the treasury stock method (the “TSM”) and, subject to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash (as defined in the Merger Agreement) amount and the receipt by Neurogene of an aggregate of $95.0 million in the Neurogene pre-closing financing. In the event Neoleukin’s net cash is below the Target Parent Net Cash amount, the exchange ratio will be adjusted such that the number of shares issued to the pre-merger Neurogene securityholders will be increased, and Neoleukin stockholders will own a smaller percentage of the combined company following the merger. In the event that Neoleukin’s net cash at closing is greater than Target Parent Net Cash amount or the aggregate proceeds from the Neurogene pre-closing financing are less than $95.0 million, the exchange ratio will be adjusted such that the number of shares issued to pre-merger Neurogene stockholders will be decreased, and Neoleukin stockholders will own a larger percentage of the combined company following the merger.

Any changes in the market price of Neoleukin stock before the completion of the merger will not affect the number of shares Neurogene stockholders will be entitled to receive pursuant to the Merger Agreement. Therefore, if before the completion of the merger, the market price of Neoleukin common stock increases from the market price on the date of the Merger Agreement, then Neurogene stockholders could receive merger consideration with substantially more value for their shares of Neurogene capital stock than the parties had negotiated when they established the exchange ratio. Similarly, if before the completion of the merger the market price of Neoleukin common stock declines from the market price on the date of the Merger Agreement, then Neurogene stockholders could receive merger consideration with substantially lower value. The Merger Agreement does not include a price-based termination right.

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Failure to complete the merger may result in either Neoleukin or Neurogene paying a termination fee to the other party, and could harm the common stock price of Neoleukin and future business and operations of each company.

If the merger is not completed, Neoleukin and Neurogene are subject to the following risks:

If the Merger Agreement is terminated and the board of directors of Neoleukin or Neurogene determines to seek another business combination, there can be no assurance that either Neoleukin or Neurogene will be able to find another third party to transact a business combination with, yielding comparable or greater benefits.

If the conditions to the merger are not satisfied or waived, the merger may not occur.

It is a condition of the consummation of the merger that the combined company stock is approved for listing on Nasdaq, but such condition can be waived by Neoleukin and Neurogene. If Nasdaq determines to delist the common stock, Neoleukin and Neurogene have not made a determination as to whether or not to waive this condition, but they could decide to waive this condition and complete the merger in such circumstance. Accordingly, there can be no assurance such listing condition will be met and, at the time you are asked to vote on the merger, you will have no assurance that securities of Neurogene Inc. will be listed on Nasdaq following the completion of the merger.

If Neoleukin and Neurogene agree to waive the requirement that the Nasdaq application be accepted for listing prior to the consummation of the merger, and their respective boards of directors determine to proceed with the closing of the merger, Nasdaq may notify the combined company of its determination to delist the combined company’s securities based upon the failure to satisfy the initial inclusion criteria. The combined company may appeal the determination to a hearings panel, which will stay the delisting action pending a panel decision. If the combined company does not appeal the determination, its common stock will be delisted. Any potential suspension of the shares of common stock from Nasdaq would likely result in decreased liquidity and increased volatility for the combined company’s common stock and would adversely affect the combined company’s ability to raise additional capital or to enter into strategic transactions. Any potential suspension of the shares of common stock from Nasdaq would also make it more difficult for stockholders to sell the combined company’s common stock in the public market.

The merger may be completed even though a material adverse effect may result from the announcement of the merger, industry-wide changes or other causes.

In general, neither Neoleukin nor Neurogene is obligated to complete the merger if there is a material adverse effect affecting the other party between July 17, 2023, the date of the Merger Agreement, and the closing of the merger. However, certain types of causes are generally excluded from the concept of a “material adverse

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If Neoleukin and Neurogene complete the merger, the combined company will need to raise additional capital by issuing equity securities or additional debt or through licensing arrangements, which may cause significant dilution to the combined company’s stockholders or restrict the combined company’s operations.

Additional financing may not be available to the combined company when it is needed or may not be available on favorable terms. To the extent that the combined company raises additional capital by issuing equity securities, such financing will cause additional dilution to all securityholders of the combined company, including Neoleukin’s pre-merger securityholders and Neurogene’s former securityholders. It is also possible that the terms of any new equity securities may have preferences over the combined company’s common stock. Any debt financing the combined company enters into may involve covenants that restrict its operations. These restrictive covenants may include limitations on additional borrowing and specific restrictions on the use of the combined company’s assets, as well as prohibitions on its ability to create liens, pay dividends, redeem its stock or make investments. In addition, if the combined company raises additional funds through licensing arrangements, it may be necessary to grant licenses on terms that are not favorable to the combined company.

Some Neoleukin and Neurogene directors and executive officers have interests in the merger that are different from yours and that may influence them to support or approve the merger without regard to your interests.

Directors and executive officers of Neoleukin and Neurogene may have interests in the merger that are different from, or in addition to, the interests of other Neoleukin stockholders generally. These interests with respect to Neoleukin’s directors and executive officers may include, among others, acceleration of stock option or restricted stock unit vesting, bonus payments, extension of exercisability periods of previously issued stock option grants, severance payments if employment is terminated in a qualifying termination in connection with the merger and rights to continued indemnification, expense advancement and insurance coverage. Two members of the Neoleukin board of directors will continue as directors of the combined company after the effective time, and, following the closing of the merger, will be eligible to be compensated as non-employee directors of the combined company. These interests with respect to Neurogene’s directors and executive officers may include, among others, the conversion of options to purchase shares of Neurogene common stock held by certain of Neurogene’s directors and executive officers into options to purchase shares of the common stock of the

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combined company at the effective time; the expected continuation of certain of Neurogene’s executive officers as executive officers of the combined company after the effective time; and the provision to all of Neurogene’s directors and executive officers of certain indemnification and liability insurance coverage pursuant to the terms of the Merger Agreement.

The Neoleukin and Neurogene boards of directors were aware of and considered those interests, among other matters, in reaching their decisions to approve and adopt the Merger Agreement, approve the merger, and recommend the approval of the Merger Agreement to Neoleukin and Neurogene stockholders. These interests, among other factors, may have influenced the directors and executive officers of Neoleukin and Neurogene to support or approve the merger.

Neoleukin stockholders and Neurogene stockholders may not realize a benefit from the merger commensurate with the ownership dilution they will experience in connection with the merger, including the conversion of Neurogene common stock issued in the Neurogene pre-closing financing.

If the combined company is unable to realize the full strategic and financial benefits currently anticipated from the merger, Neoleukin stockholders and Neurogene stockholders will have experienced substantial dilution of their ownership interests without receiving any commensurate benefit, or only receiving part of the commensurate benefit to the extent the combined company is able to realize only part of the strategic and financial benefits currently anticipated from the merger.

If the merger is not completed, Neoleukin’s stock price may decline significantly.

The market price of Neoleukin common stock is subject to significant fluctuations. Market prices for securities of pharmaceutical, biotechnology and other life science companies have historically been particularly volatile. In addition, the market price of Neoleukin common stock will likely be volatile based on whether stockholders and other investors believe that Neoleukin can complete the merger or otherwise raise additional capital to support Neoleukin’s operations if the merger is not consummated and another strategic transaction cannot be identified, negotiated and consummated in a timely manner, or at all. The volatility of the market price of Neoleukin common stock has been and may be exacerbated by low trading volume. Additional factors that may cause the market price of Neoleukin common stock to fluctuate include:

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Moreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual companies. These broad market fluctuations may also adversely affect the trading price of Neoleukin common stock. In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against such companies.

Neoleukin and Neurogene securityholders will generally have a reduced ownership and voting interest in, and will exercise less influence over the management of, the combined company following the completion of the merger as compared to their current ownership and voting interests in the respective companies.

After the completion of the merger, the current stockholders of Neoleukin and Neurogene will generally own a smaller percentage of the combined company than their ownership of their respective companies prior to the merger. Immediately after the merger, Neoleukin stockholders as of immediately prior to the merger are expected to own approximately 16% of the outstanding shares of capital stock of the combined company and former Neurogene securityholders, including shares of Neurogene common stock and Neurogene pre-funded warrants purchased in the Neurogene pre-closing financing, are expected to own approximately 84% of the outstanding shares of capital stock of the combined company, using the TSM and subject to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash amount and the receipt of aggregate proceeds of $95.0 million from the Neurogene pre-closing financing. As a result, some stockholders will hold a smaller pro rata share and, therefore, a lower percentage of the voting stock of the combined company than such stockholder currently holds in Neoleukin or Neurogene as a stand-alone company, respectively.

During the pendency of the merger, each of Neoleukin and Neurogene may be limited in its ability to enter into a business combination with another party on more favorable terms because of restrictions in the Merger Agreement, which could adversely affect their respective business prospects.

Certain provisions of the Merger Agreement may discourage third parties from submitting competing proposals, including proposals that may be superior to the transactions contemplated by the Merger Agreement.

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Because the lack of a public market for Neurogene’s common stock makes it difficult to evaluate the fair market value of Neurogene’s capital stock, the value of the Neoleukin common stock to be issued to Neurogene stockholders may be more or less than the fair market value of Neurogene’s common stock.

The outstanding capital stock of Neurogene is privately held and is not traded in any public market. The lack of a public market makes it difficult to determine the fair market value of Neurogene’s capital stock. Because the percentage of Neoleukin equity to be issued to Neurogene stockholders was determined based on negotiations between the parties, it is possible that the value of the Neoleukin common stock to be issued to Neurogene stockholders will be more or less than the fair market value of Neurogene’s capital stock.

If the merger does not qualify as a reorganization under Section 368(a) of the Internal Revenue Code of 1986, as amended (the “Code”), U.S. holders of Neurogene common stock may be taxed on the full amount of the consideration received in the merger.

As discussed more fully under the section entitled “The Merger—Material U.S. Federal Income Tax Considerations of the Merger,” the merger is intended to qualify for U.S. federal income tax purposes as a “reorganization” within the meaning of Section 368(a) of the Code. Assuming the merger so qualifies, no gain will be recognized by U.S. holders (as defined in the section entitled “The Merger—Material U.S. Federal Income Tax Considerations of the Merger”) of Neurogene common stock who receive only Neoleukin common stock in the merger. It is not, however, a condition to Neurogene’s obligation or Neoleukin’s obligation to complete the transactions that the merger so qualifies. Neurogene and Neoleukin have not sought and will not seek any ruling from the IRS regarding any matters relating to the transactions and, as a result, there can be no assurance that the IRS would not assert, or that a court would not sustain, a position contrary to any of the conclusions set forth herein. If the merger does not qualify for the U.S. federal income tax treatment described herein, U.S. holders of Neurogene common stock may be taxed on any gain realized up to the full fair market value of any Neoleukin common stock received in the merger.

The tax treatment of the Contingent Value Rights (“CVRs”) is uncertain.

There is no authority directly on point addressing whether contingent value rights with characteristics similar to the CVRs should be treated for federal income tax purposes as a distribution of property with respect to its stock, an “open transaction,” or in some other manner, and such questions are inherently factual in nature. Accordingly, holders are urged to consult with their tax advisors regarding this issue.

However, based on the specific characteristics of the CVRs, and unless otherwise required by a change in law after the date of the CVR Agreement (as defined below), Neoleukin intends to take the position that the fair market value of the CVRs cannot be reasonably ascertained on the date of the issuance of the CVRs and, accordingly, the issuance of the CVRs constitutes an “open transaction.” Unless otherwise required by a change in law or the good-faith resolution of a tax controversy, Neoleukin will not report the issuance of the CVRs as a current distribution of property with respect to its stock and will instead report each future cash payment (if any) on the CVRs as a distribution by Neoleukin for U.S. federal income tax purposes, with each such payment being reported as a dividend to the extent of Neoleukin’s current or accumulated earnings and profits in the year in which such payment is made.

If Neoleukin’s intended reporting position is correct, a U.S. holder (as defined in the section entitled “The Merger—Material U.S. Federal Income Tax Considerations of the Merger”) would not generally recognize income in respect of the CVRs at the time they are distributed and would take no tax basis in the CVRs. Any future cash payments would constitute a dividend to the extent of Neoleukin’s current or accumulated earnings and profits (as determined for U.S. federal income tax purposes) in the taxable year of such payment, then as a non-taxable return of capital to the extent of such holder’s basis in its Neoleukin common stock, and finally as capital gain from the sale or exchange of Neoleukin common stock. Dividends received by individual U.S. holders are eligible for reduced rates of taxation applicable to long-term capital gains, provided certain holding period requirements are met.

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It is possible that the IRS could instead assert that the issuance of the CVRs should be treated as a “closed transaction.” Under “closed transaction” treatment, a U.S. holder would be treated as receiving a distribution equal to the fair market value of the CVRs at the time they are issued to such U.S. holder. The amount of this distribution generally would be treated first as a taxable dividend to the extent of the U.S. holder’s pro rata share of Neoleukin’s current or accumulated earnings and profits (as determined for U.S. federal income tax purposes), then as a non-taxable return of capital to the extent of the U.S. holder’s basis in its Neoleukin common stock, and finally as capital gain from the sale or exchange of Neoleukin common stock. A U.S. holder’s tax basis in the CVRs received would equal the fair market value of the CVRs at the time they are issued and the holding period of the CVRs received would begin on the day following the date they are issued. Although not free from doubt, a future cash payment under a CVR would likely be treated as a non-taxable return of a U.S. holder’s adjusted tax basis in the CVR to the extent thereof, although the timing of the recovery of a U.S. holder’s tax basis is unclear. A payment in excess of such amount may be treated as a payment with respect to a sale of a capital asset, ordinary income or dividends. Additionally, it is possible that a portion of future cash payments would constitute imputed interest and taxed as such. A U.S. holder might recognize loss, which might be a capital loss and could be a long-term capital loss, upon the expiration of the CVR to the extent cash payments ultimately received pursuant to such CVR were less than the U.S. holder’s adjusted tax basis in the CVRs, but whether and when such a loss would be recognized is unclear. The deductibility of capital losses is subject to limitations.

It is possible, although Neoleukin believes unlikely, that the issuance of the CVRs could be treated as one or more “debt instruments” or as a distribution of equity.

U.S. holders are urged to consult their tax advisors with respect to the proper characterization of the CVRs and the tax considerations thereof (including any future cash payments made under the CVRs).

Risks Related to the Proposed Reverse Stock Split

The reverse stock split may not increase the combined company’s stock price over the long-term.

The principal purposes of the reverse stock split are to (i) increase the per-share market price of Neoleukin’s common stock above the minimum bid price requirement under the Nasdaq rules so that the listing of Neoleukin as the combined company and the shares of Neoleukin common stock being issued in the merger on Nasdaq will be approved and (ii) increase the number of authorized and unissued shares available for future issuance in connection with the merger. It cannot be assured, however, that the reverse stock split will accomplish any increase in the per-share market price of the combined company’s common stock for any meaningful period of time. While it is expected that the reduction in the number of outstanding shares of common stock will proportionally increase the market price of the combined company’s common stock, it cannot be assured that the reverse stock split will increase the market price of its common stock by a multiple of the reverse stock split ratio mutually agreed by Neoleukin and Neurogene, or result in any permanent or sustained increase in the market price of Neoleukin’s common stock, which is dependent upon many factors, including the combined company’s business and financial performance, general market conditions and prospects for future success. Thus, while the stock price of the combined company might meet the listing requirements for Nasdaq initially after the reverse stock split, it cannot be assured that it will continue to do so.

The reverse stock split may decrease the liquidity of the combined company’s common stock.

Although the Neoleukin board of directors believes that the anticipated increase in the market price of the combined company’s common stock resulting from the proposed reverse stock split could encourage interest in its common stock and possibly promote greater liquidity for its stockholders, such liquidity could also be adversely affected by the reduced number of shares outstanding after the reverse stock split. The reduction in the number of outstanding shares may lead to reduced trading and a smaller number of market makers for the combined company’s common stock. In addition, the reverse stock split may not result in an increase in the combined company’s stock price necessary to satisfy Nasdaq’s initial listing requirements for the combined company.

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The reverse stock split may lead to a decrease in the combined company’s overall market capitalization.

Should the market price of the combined company’s common stock decline after the reverse stock split, the percentage decline may be greater, due to the smaller number of shares outstanding, than it would have been prior to the reverse stock split. A reverse stock split is often viewed negatively by the market and, consequently, can lead to a decrease in the combined company’s overall market capitalization. If the per share market price does not increase in proportion to the reverse stock split ratio, then the value of the combined company, as measured by its stock capitalization, will be reduced. In some cases, the per-share stock price of companies that have effected reverse stock splits subsequently declined back to pre-reverse split levels, and accordingly, it cannot be assured that the total market value of the combined company’s common stock will remain the same after the reverse stock split is effected, or that the reverse stock split will not have an adverse effect on the combined company’s stock price due to the reduced number of shares outstanding after the reverse stock split.

Risks Related to Neoleukin’s Strategic Alternative Process and Potential Strategic Transaction

Failure to complete, or delays in completing, the proposed merger transaction with Neurogene could materially and adversely affect Neoleukin’s results of operations, business, financial results and/or stock price.

In March 2023, Neoleukin announced that it intended to conduct a comprehensive review of strategic alternatives for Neoleukin and its assets. After a comprehensive review of strategic alternatives, including identifying and reviewing potential candidates for the merger, on July 17, 2023, Neoleukin entered into the Merger Agreement with Neurogene and Merger Sub, pursuant to which, subject to the satisfaction or waiver of the conditions therein, Merger Sub will merge with and into Neurogene, with Neurogene surviving as a wholly owned subsidiary of Neoleukin. The closing of the merger is subject to approval by the stockholders of Neoleukin and Neurogene as well as other customary closing conditions, including the effectiveness of a registration statement filed with the SEC in connection with the transaction. If the merger is completed, the business of Neurogene will continue as the business of the combined company. Any failure to satisfy a required condition to closing may prevent, delay or otherwise materially and adversely affect the completion of the transaction, which could materially and adversely affect Neoleukin’s results of operations, business, financial results and/or stock price. Neoleukin cannot predict with certainty whether or when any of the required closing conditions will be satisfied or if another uncertainty may arise and cannot assure you that the proposed merger will be successfully consummated or that Neoleukin will be able to successfully consummate the proposed merger as currently contemplated under the Merger Agreement or at all.

Neoleukin’s efforts to complete the merger could cause substantial disruptions in, and create uncertainty surrounding, Neoleukin’s business, which may materially adversely affect Neoleukin’s results of operations and Neoleukin’s business. Uncertainty as to whether the merger will be completed may affect Neoleukin’s ability to retain and motivate existing employees or recruit prospective employees if vacancies in staffing need to be filled. Employee retention may be particularly challenging while the transaction is pending because employees may experience uncertainty about their roles following the transaction. A substantial amount of Neoleukin’s management’s and employees’ attention is being directed toward the completion of the transaction and thus is being diverted from Neoleukin’s day-to-day operations. Uncertainty as to Neoleukin’s future could adversely affect Neoleukin’s business and Neoleukin’s relationship with suppliers, vendors, regulators and other business partners. For example, vendors and other counterparties may defer decisions about working with Neoleukin or seek to change existing business relationships with Neoleukin. Changes to, or termination of, existing business relationships could adversely affect Neoleukin’s results of operations and financial condition, as well as the market price of Neoleukin’s common stock. The adverse effects of the pendency of the transaction could be exacerbated by any delays in completion of the transaction or termination of the Merger Agreement.

Risks related to the failure to consummate, or delay in consummating, the proposed merger transaction with Neurogene include, but are not limited to, the following:

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The occurrence of any of these events individually or in combination could materially and adversely affect Neoleukin’s results of operations, business and stock price.

Neoleukin cannot be sure if or when the merger will be completed.

The consummation of the merger is subject to the satisfaction or waiver of various conditions, including the authorization of the merger by Neoleukin’s stockholders and Neurogene’s stockholders. Neoleukin cannot guarantee that the closing conditions set forth in the Merger Agreement will be satisfied. If Neoleukin is unable to satisfy certain closing conditions or if other mutual closing conditions are not satisfied, Neurogene will not be obligated to complete the merger. Under certain circumstances, Neoleukin would be required to pay Neurogene a termination fee of $3.04 million, and/or expense reimbursement of Neurogene of up to $1.0 million.

If the merger is not completed, Neoleukin’s board of directors, in discharging its fiduciary obligations to Neoleukin’s stockholders, would evaluate other strategic alternatives or financing options that may be available, which alternatives may not be as favorable to Neoleukin’s stockholders as the merger, including a liquidation and dissolution. Any future sale or merger, financing or other transaction, including a liquidation or dissolution, may be subject to further stockholder approval. Neoleukin may also be unable to find, evaluate or complete other strategic alternatives, which may have a materially adverse effect on Neoleukin’s business.

Until the merger is completed, the Merger Agreement restricts Neoleukin from taking specified actions without the consent of Neurogene, and requires Neoleukin to operate in the ordinary course of business consistent with past practice. These restrictions may prevent Neoleukin from making appropriate changes to its business or pursuing attractive business opportunities that may arise prior to the completion of the merger. Further, if Neoleukin’s net cash at closing is lower than anticipated, either because expenses exceed current estimates or due to delays prior to closing, then the pre-merger stockholders of Neoleukin will own less of the combined company pursuant to the exchange ratio adjustment set forth in the Merger Agreement. Neoleukin maintaining a minimum of the net cash amount is a condition to closing the merger.

Any delay in completing the proposed merger may materially and adversely affect the timing and benefits that are expected to be achieved from the proposed merger.

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The exchange ratio set forth in the Merger Agreement is not adjustable based on the market price of Neoleukin’s common stock, so the merger consideration at the closing of the merger may have a greater or lesser value than at the time the Merger Agreement was signed.

The Merger Agreement has set the exchange ratio for Neurogene capital stock being converted into Neoleukin’s common stock, and the exchange ratio is based on the relative value of Neurogene and Neoleukin as determined pursuant to the Merger Agreement, in each case immediately prior to the closing of the merger. Assuming Neoleukin has Target Parent Net Cash at closing and Neurogene receives the expected $95.0 million in aggregate proceeds from the Neurogene pre-closing financing, applying the exchange ratio formula in the Merger Agreement, the former Neurogene securityholders immediately before the merger are expected to own approximately 84% of outstanding capital stock of the combined company immediately following the merger, and the securityholders of Neoleukin immediately before the merger are expected to own approximately 16% of outstanding capital stock of the combined company immediately following the merger, in each case, calculated using the TSM and after giving effect to the Neurogene pre-closing financing and subject to certain assumptions detailed in the Merger Agreement. Under certain circumstances further described in the Merger Agreement, however, these ownership percentages may be adjusted upward or downward based on the net cash held by Neoleukin at the closing of the merger and the aggregate proceeds of the Neurogene pre-closing financing, and as a result, either the Neoleukin stockholders or the Neurogene stockholders could own less of the combined company than expected.

Any changes in the market price of Neoleukin’s common stock before the completion of the merger will not affect the number of shares of Neoleukin’s common stock issuable to Neurogene’s stockholders pursuant to the Merger Agreement. Therefore, if before the completion of the merger the market price of Neoleukin’s common stock declines from the market price on the date of the Merger Agreement, then Neurogene’s stockholders could receive merger consideration with substantially lower value than the value of such merger consideration on the date of the Merger Agreement. Similarly, if before the completion of the merger the market price of Neoleukin’s common stock increases from the market price of Neoleukin’s common stock on the date of the Merger Agreement, then Neurogene’s stockholders could receive merger consideration with substantially greater value than the value of such merger consideration on the date of the Merger Agreement. The Merger Agreement does not include a price-based termination right.

The Merger Agreement contains provisions that limit Neoleukin’s ability to pursue alternatives to the merger, could discourage a potential competing acquiror of Neoleukin from making an alternative transaction proposal and, in specified circumstances, could require Neoleukin to pay a termination fee to Neurogene, which could significantly harm Neoleukin’s financial condition and the market price of Neoleukin’s common stock and negatively affect the future business and operations of each company.

The Merger Agreement contains provisions that make it difficult for Neoleukin to entertain a third-party proposal for an acquisition of Neoleukin. These provisions include Neoleukin’s agreement not to solicit or initiate any additional discussions with third parties regarding other proposals for Neoleukin’s acquisition, as well as restrictions on Neoleukin’s ability to respond to such proposals, subject to fulfillment of certain fiduciary requirements of Neoleukin’s board of directors.

If the proposed merger is not completed and the Merger Agreement is terminated under certain circumstances, Neoleukin may be required to pay Neurogene a termination fee of up to $3.04 million, and/or expense reimbursement of up to $1.0 million. Even if a termination fee is not payable in connection with a termination of the Merger Agreement, Neoleukin will have incurred significant fees and expenses, which must be paid whether or not the merger is completed. Further, if the proposed merger is not completed, it could significantly harm the market price of Neoleukin’s common stock.

In addition, if the Merger Agreement is terminated and the board of directors of Neoleukin determines to seek another business combination, there can be no assurance that Neoleukin will be able to find a partner or, if such partner is found, to close an alternative transaction on terms that are as favorable or more favorable than the terms set forth in the Merger Agreement.

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Litigation has arisen, and more could arise, in connection with the Merger, against Neoleukin or Neurogene which could be costly, prevent consummation of the merger, divert management’s attention and otherwise materially harm Neoleukin’s, Neurogene’s or the combined company’s business.

A putative stockholder complaint has been filed, and additional putative stockholder complaints, including stockholder class action complaints, and other complaints may be filed against Neoleukin, Neoleukin’s board of directors, Neurogene, Neurogene’s board of directors and others in connection with the transactions contemplated by the Merger Agreement. The outcome of litigation is uncertain, and Neoleukin or Neurogene may not be successful in defending against any such existing or future claims. Lawsuits filed against Neoleukin, Neoleukin’s board of directors, Neurogene, or Neurogene’s board of directors could delay or prevent the merger, divert the attention of Neoleukin’s management and employees from Neoleukin’s day-to-day business and otherwise adversely affect Neoleukin’s financial condition.

Neoleukin’s stockholders potentially may not receive any payment on the CVRs and the CVRs may otherwise expire valueless.

Neoleukin may not be able to achieve successful results from the reduction in liabilities of lease obligations and the disposition of such assets as described above, and may not receive a material amount of money from any sales tax refund that may be issued to Neoleukin. Further, if none of these are achieved for any reason within the time periods specified in the CVR Agreement, no payments will be made under the CVRs, and the CVRs will expire valueless.

Certain of Neoleukin’s officers and directors may have interests in the proposed merger that are different from, or in conflict with or in addition to, those of Neoleukin’s stockholders generally.

Certain officers and directors of Neoleukin may have interests in the proposed merger that are different from the interests of Neoleukin’s stockholders generally, including potentially, among others, the continued service as a director of the combined company, the acceleration of stock option or restricted stock unit (“RSU”) vesting, and continued indemnification.

The closing of the merger will also result in the acceleration of vesting of certain options to purchase shares of Neoleukin’s common stock and RSUs held by Neoleukin’s executive officers and directors, whether or not there is a covered termination of such officer’s employment or board membership. In addition, two of Neoleukin’s current directors are expected to become directors of the surviving company upon the closing of the merger, and all of Neoleukin’s directors and executive officers are entitled to certain indemnification and liability insurance coverage pursuant to the terms of the Merger Agreement. These interests, among others, may influence the officers and directors of Neoleukin and cause them to view the merger differently from how Neoleukin’s stockholders generally may view it.

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Neoleukin’s equityholders will have a reduced ownership and voting interest in, and will exercise less influence over the management of the combined company, Neoleukin following the closing of the merger as compared to their current ownership and voting interest in Neoleukin.

After the completion of the merger, the current securityholders of Neoleukin will own a smaller percentage of the combined company than their ownership in Neoleukin prior to the merger. Immediately after the merger, it is currently estimated that pre-merger Neoleukin’s equityholders will own approximately 16% of the common stock of the combined company, and pre-merger Neurogene equityholders will own approximately 84% of the common stock of the combined company, in each case, calculated using the TSM and after giving effect to the Neurogene pre-closing financing and subject to certain assumptions. These estimates are based on the anticipated exchange ratio and are subject to adjustment as provided in the Merger Agreement.

In addition, the board of directors of the combined company, which is expected to have a total of seven directors immediately following the closing of the merger, will initially include two individuals with prior affiliations with Neoleukin. Consequently, securityholders of Neoleukin will not be able to exercise the same influence over the management and policies of the combined organization following the closing of the merger than they currently exercise over the management and policies of Neoleukin.

Neoleukin’s stockholders may not realize a benefit from the merger commensurate with the ownership dilution they will experience in connection with the merger.

If the combined company is unable to realize the strategic and financial benefits currently anticipated from the proposed merger, Neoleukin’s stockholders will have experienced substantial dilution of their ownership interests in Neoleukin without receiving the expected commensurate benefit, or only receive part of the commensurate benefit to the extent the combined company is able to realize only part of the expected strategic and financial benefits currently anticipated from the proposed merger.

If Neoleukin does not successfully consummate the merger or another strategic transaction, Neoleukin’s board of directors may decide to pursue a dissolution and liquidation of Neoleukin. In such an event, the amount of cash available for distribution to Neoleukin’s stockholders will depend heavily on the costs and timing of such liquidation as well as the amount of cash that will need to be reserved for commitments and contingent liabilities, as to which Neoleukin can give you no assurance.

There can be no assurance that the merger will be completed. If the merger is not completed, Neoleukin’s board of directors may decide to pursue a dissolution and liquidation of Neoleukin. In such an event, the amount of cash available for distribution to Neoleukin’s stockholders will depend heavily on the costs related to and timing of such decision and, ultimately, such liquidation, since the amount of cash available for distribution continues to decrease as Neoleukin funds its operations while pursuing the merger. In addition, if Neoleukin’s board of directors were to approve and recommend, and Neoleukin’s stockholders were to approve, a dissolution and liquidation of Neoleukin, Neoleukin would be required under Delaware corporate law to pay Neoleukin’s outstanding obligations, as well as to make reasonable provision for contingent and unknown obligations, prior to making any distributions in liquidation to stockholders. Neoleukin’s commitments and contingent liabilities may include obligations under Neoleukin’s employment and related agreements with certain employees that provide for severance and other payments following a termination of employment occurring for various reasons,

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including a change in control of Neoleukin, which may include a dissolution of Neoleukin, litigation against Neoleukin, and other various claims and legal actions arising in the ordinary course of business, and other unexpected and/or contingent liabilities. As a result of this requirement, a portion of Neoleukin’s assets would need to be reserved pending the resolution of such obligations.

In addition, Neoleukin may be subject to litigation or other claims related to a dissolution and liquidation of Neoleukin. If a dissolution and liquidation were to be pursued, Neoleukin’s board of directors, in consultation with Neoleukin’s advisors, would need to evaluate these matters and make a determination about a reasonable amount to reserve. Accordingly, holders of Neoleukin’s common stock could lose all or a significant portion of their investment in the event of liquidation, dissolution or winding up of Neoleukin. A liquidation would be a lengthy and uncertain process with no assurance of any value ever being returned to Neoleukin’s stockholders.

Neoleukin is substantially dependent on Neoleukin’s remaining employees to facilitate the consummation of the merger.

Neoleukin’s ability to consummate a strategic transaction depends upon its ability to retain its employees required to consummate such a transaction, the loss of whose services may adversely impact the ability to consummate such transaction. In November 2022, and then again in March 2023, Neoleukin undertook an organizational restructuring that significantly reduced its workforce in order to conserve its capital resources. As of September 18, 2023, Neoleukin had only 7 full-time employees. Neoleukin’s ability to successfully complete the merger depends in large part on Neoleukin’s ability to retain certain remaining personnel. Despite Neoleukin’s efforts to retain these employees, one or more may terminate their employment with Neoleukin on short notice. Neoleukin’s cash conservation activities may yield other unintended consequences, such as attrition beyond its planned reduction in workforce and reduced employee morale, which may cause remaining employees to seek alternative employment. The loss of the services of certain employees could potentially harm Neoleukin’s ability to consummate the merger, to run Neoleukin’s day-to-day business operations, as well as to fulfill Neoleukin’s reporting obligations as a public company.

Risks Related to Neurogene

Risks Related to Neurogene’s Limited Operating History, Financial Position and Capital Requirements

Neurogene has a limited operating history, has not completed any clinical trials, and has no products approved for commercial sale, which may make it difficult for you to evaluate its current business and likelihood of success and viability.

Neurogene is a clinical-stage biotechnology company with limited operating history. Since its inception in 2018, Neurogene has incurred significant operating losses and has utilized substantially all of its resources to conduct research and development activities (including with respect to its NGN-401 and NGN-101 programs), preclinical studies and clinical trials of Neurogene’s most advanced product candidates, establish in-house manufacturing capabilities, including analytical and process development operations to support ongoing manufacturing operations, manufacture product candidates, conduct business planning, develop and maintain its intellectual property portfolio, hire personnel, raise capital, and provide general and administrative support for these activities. Neurogene has little experience as a company in initiating, conducting or completing clinical trials. In part because of this lack of experience, Neurogene cannot be certain that its current and planned clinical trials will begin or be completed on time, if at all. In addition, while Neurogene is conducting a Phase 1/2 clinical trial of NGN-401 in patients with Rett syndrome and a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease, Neurogene has not yet demonstrated its ability to successfully complete clinical trials (including Phase 3 or other pivotal clinical trials), obtain regulatory or marketing approvals, manufacture a commercial-scale product or arrange for a third party to do so on its behalf, or conduct sales, marketing and distribution activities necessary for successful product commercialization. Additionally, Neurogene expects its financial condition and operating results to continue to fluctuate significantly from period to period due to a variety of factors, many of which are beyond its control. Consequently, any predictions made about Neurogene’s future success or viability may not be as accurate as they could be if Neurogene had a longer operating history.

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In addition, as its business grows, Neurogene may encounter unforeseen expenses, restrictions, difficulties, complications, delays and other known and unknown factors. Neurogene will need to transition at some point from a company with an early research and development focus to a company capable of supporting larger pivotal clinical trials and eventually commercial activities, including the manufacture of commercial scale product. Neurogene may not be successful in such a transition.

Even if the merger and pre-closing financing are successful, Neurogene will require substantial additional capital to finance its operations in the future. If Neurogene is unable to raise such capital when needed, or on acceptable terms, Neurogene may be forced to delay, reduce or eliminate clinical trials, product development programs or future commercialization efforts.

Developing biotechnology products is a long, time-consuming, expensive and uncertain process that takes years to complete. Since its inception, Neurogene has funded its operations primarily through private financings and has incurred significant recurring losses, including net losses of $55.2 million and $50.5 million for the years ended December 31, 2022 and 2021, respectively. Neurogene expects its expenses to increase in connection with its ongoing activities, particularly as Neurogene conducts a Phase 1/2 clinical trial of NGN-401 in patients with Rett syndrome and a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease and initiates additional clinical trials, and continues to research, develop and conduct preclinical studies of its other potential product candidates. In addition, if Neurogene obtains regulatory approval for any product candidate for commercial sale, including NGN-401 and NGN-101, Neurogene anticipates incurring significant commercialization expenses related to product manufacturing, marketing, sales and distribution activities to launch any such product. Neurogene’s expenses could increase beyond expectations if Neurogene is required by the FDA or other regulatory agencies to perform preclinical studies or clinical trials in addition to those that Neurogene currently anticipates. Because the design and outcome of its current, planned and anticipated clinical trials are highly uncertain, Neurogene cannot reasonably estimate the actual amount of funding that will be necessary to successfully complete the development and commercialization of any product candidate Neurogene develops. Neurogene’s future capital requirements depend on many factors, including factors that are not within its control.

Following the merger and pre-closing financing, Neurogene will also incur additional costs associated with operating as a public company. Accordingly, Neurogene will require substantial additional funding to continue its operations. Based on its current operating plan, and assuming the merger and pre-closing financing are successfully completed, Neurogene believes that its existing cash, cash equivalents and short-term investments should be sufficient to fund its operations into the second half of 2026. This estimate is based on assumptions that may prove to be materially wrong, and Neurogene could deplete its available capital resources sooner than it currently expects. Neurogene’s future capital requirements will depend on many factors, including:

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Neurogene does not have any committed external sources of funds and adequate additional financing may not be available to it on acceptable terms, or at all. Neurogene may be required to seek additional funds sooner than planned through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources. Such financing may dilute its stockholders or the failure to obtain such financing may restrict its operating activities. Any additional fundraising efforts may divert Neurogene’s management from their day-to-day activities, which may adversely affect its business. To the extent that Neurogene raises additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms may include liquidation or other preferences and anti-dilution protections that adversely affect your rights as a stockholder. Debt financing may result in imposition of debt covenants, increased fixed payment obligations or other restrictions that may affect Neurogene’s business. If Neurogene raises additional funds through upfront payments or milestone payments pursuant to future collaborations with third parties, Neurogene may have to relinquish valuable rights to product development programs, or grant licenses on terms that are not favorable to it. Neurogene’s ability to raise additional capital may be adversely impacted by global macroeconomic conditions and volatility in the credit and financial markets in the United States and worldwide, over which Neurogene may have no or little control. Its failure to raise capital as and when needed or on acceptable terms would have a negative impact on its financial condition and its ability to pursue its business strategy, and Neurogene may have to delay, reduce the scope of, suspend or eliminate clinical trials, product development programs or future commercialization efforts.

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Neurogene has incurred significant losses since inception, and expects to incur significant losses for the foreseeable future and may not be able to achieve or sustain profitability in the future. Neurogene has no products for sale, has not generated any product revenue and may never generate product revenue or become profitable.

Investment in biotechnology product development is a highly speculative undertaking and entails substantial upfront expenditures and significant risks that any program will fail to demonstrate adequate efficacy or potency or an acceptable safety profile, gain regulatory approval and become commercially viable. Neurogene has no products approved for commercial sale, has not generated any revenue from product sales to date, and continues to incur significant research and development and other expenses related to its ongoing operations. Neurogene does not expect to generate product revenue unless or until Neurogene successfully completes clinical development and obtains regulatory approval of, and then successfully commercializes, at least one product candidate. Neurogene may never succeed in these activities and, even if it does, may never generate product revenue or revenues that are significant or large enough to achieve profitability. If Neurogene is unable to generate sufficient revenue through the sale of any approved products, it may be unable to continue operations without additional funding.

Neurogene has incurred significant net losses in each period since it commenced operations in 2018. Neurogene’s net loss was $55.2 million for the year ended December 31, 2022. Neurogene expects to continue to incur significant losses for the foreseeable future. Its operating expenses and net losses may fluctuate significantly from quarter to quarter and year to year. Neurogene anticipates that its expenses will increase substantially if and as it:

In addition, Neurogene’s expenses will increase if, among other things, it is required by the FDA or other regulatory authorities to perform trials or studies in addition to, or different than, those that Neurogene currently anticipates, there are any delays in completing its clinical trials or the development of any product candidates, or there are any third-party challenges to its intellectual property or Neurogene needs to defend against any intellectual property-related claim.

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Even if Neurogene obtains marketing approval for, and is successful in commercializing, one or more product candidates, Neurogene expects to incur substantial additional research and development and other expenditures to develop and market additional programs and/or to expand the approved indications of any marketed product. Neurogene may encounter unforeseen expenses, difficulties, complications, delays and other unknown factors that may adversely affect its business. The size of its future net losses will depend, in part, on the rate of future growth of its expenses and its ability to generate revenue.

Neurogene’s failure to become profitable would decrease the value of Neurogene and could impair its ability to raise capital, maintain its research and development efforts, expand its business and/or continue its operations. A decline in the value of Neurogene could also cause you to lose all or part of your investment.

In addition, management have evaluated adverse conditions and events that raise substantial doubt about Neurogene’s ability to continue as a going concern, and, as a result, its independent registered public accounting firm included an explanatory paragraph in its report on its financial statements as of and for the year ended December 31, 2022 included elsewhere herein with respect to this uncertainty. This going concern opinion could materially limit Neurogene’s ability to raise additional funds through the issuance of new debt or equity securities or otherwise. Future reports on its financial statements may include an explanatory paragraph with respect to its ability to continue as a going concern. Even if the merger and pre-closing financing are successfully completed, there is no assurance that adequate additional financing needed to allow Neurogene to continue as a going concern will be available to Neurogene on acceptable terms, or at all. The perception that Neurogene may not be able to continue as a going concern may cause others to choose not to do business with Neurogene due to concerns about its ability to meet its contractual obligations.

Risks Related to Discovery, Development and Commercialization

Neurogene faces competition from entities that have developed or may develop programs for the diseases it plans to address with NGN-401 and NGN-101 or other product candidates.

The development and commercialization of biological products is highly competitive. If approved, NGN-401 and NGN-101 or other product candidates will face significant competition and Neurogene’s failure to effectively compete may prevent it from achieving significant market penetration. Neurogene competes with a variety of multinational biopharmaceutical companies, specialized biotechnology companies and emerging biotechnology companies, as well as academic institutions, governmental agencies, and public and private research institutions, among others. Many of the companies with which Neurogene is currently competing or will compete against in the future have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals, and marketing approved products than Neurogene does. Mergers and acquisitions in the pharmaceutical and biotechnology industry may result in even more resources being concentrated among a smaller number of its competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with Neurogene in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sites, patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, NGN-401 and NGN-101 or other product candidates.

As described in the section entitled “Neurogene’s Business—Competition”, Neurogene’s competitors have developed, are developing or may develop programs or clinical stage products competitive with NGN-401 or NGN-101 or other earlier stage product candidates. Competitive therapeutic treatments include those that have already been approved and accepted by the medical community and any new treatments for Rett syndrome or for CLN5 Batten disease. Neurogene’s success will depend partially on its ability to develop and commercialize products that have a competitive safety, efficacy or potency, dosing and/or presentation profile. Neurogene’s commercial opportunity and success will be reduced or eliminated if competing products are safer, more effective or potent, have a more attractive or less invasive dosing profile or presentation or are less expensive

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than any products Neurogene may develop, if any, or if competitors develop competing products or if biosimilars enter the market more quickly than Neurogene is able to, if at all, and are able to gain market acceptance.

NGN-401, NGN-101 and Neurogene’s other programs are in early stages of development and may fail in development or suffer delays that materially and adversely affect their commercial viability. If Neurogene or its current or future collaborators are unable to complete development of, or commercialize, Neurogene’s product candidates, or experience significant delays in doing so, its business will be materially harmed.

Neurogene has no products on the market and NGN-401 and NGN-101 are in the early stages of clinical development, while Neurogene’s other programs are in early stages of preclinical development. As a result, Neurogene expects it will be many years before it commercializes these product candidates and ultimately may not be successful in commercializing any of its product candidates. Neurogene’s ability to achieve and sustain profitability depends on obtaining regulatory approvals for, and successfully commercializing, its lead product candidate NGN-401 or other product candidates, including NGN-101, either alone or with third parties, and Neurogene cannot guarantee that it will ever obtain regulatory approval for any product candidates. Neurogene has limited experience as a company in conducting and managing the clinical trials necessary to obtain regulatory approvals, including approval by the FDA or comparable foreign regulatory authorities. Neurogene has also not yet demonstrated its ability to obtain regulatory approvals, manufacture a commercial scale product or arrange for a third party to do so on its behalf, or conduct sales and marketing activities necessary for successful product commercialization. Before obtaining regulatory approval for the commercial distribution of product candidates, Neurogene or an existing or future collaborator must conduct extensive preclinical tests and clinical trials to demonstrate the safety, purity and efficacy or potency in humans of such product candidates.

Neurogene or its collaborators may experience delays in initiating or completing clinical trials, and also may experience unforeseen events during, or as a result of, any current or future clinical trials that could delay or prevent its ability to receive marketing approval or commercialize NGN-401 or NGN-101 or any other product candidates, including:

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Commencing clinical trials in the United States is subject to acceptance by the FDA of an IND or, if commenced in other jurisdictions, acceptance by the comparable foreign regulatory agency of a similar application, as well as finalizing the trial design. In the event that the FDA or applicable foreign regulatory agency requires Neurogene to complete additional preclinical studies, or Neurogene is required to satisfy other regulatory requests prior to commencing clinical trials, the start of its clinical trials may be delayed. Even after Neurogene receives and incorporates guidance from these regulatory authorities, the FDA or other regulatory authorities could disagree that Neurogene has satisfied their requirements to commence any clinical trial or change their position on the acceptability of its trial design or the clinical endpoints selected, which may require Neurogene to complete additional preclinical studies or clinical trials, delay the enrollment of its clinical trials or impose stricter approval conditions than Neurogene currently expects. There are equivalent processes and risks applicable to clinical trial applications in other jurisdictions, including the United Kingdom (“UK”), Australia and the European Union.

Neurogene may not have the financial resources to continue development of, or to modify existing collaborations or enter into new collaborations for, a product candidate if Neurogene experiences any issues that delay or prevent regulatory approval of, or its ability to commercialize, NGN-401 or NGN-101 or any other product candidates. Neurogene or its current or future collaborators’ inability to complete development of, or commercialize, NGN-401 or NGN-101 or any other product candidates or significant delays in doing so, could have a material and adverse effect on its business, financial condition, results of operations and prospects.

Neurogene currently utilizes adeno-associated virus serotype 9 (“AAV9”) capsid for delivery of therapeutic transgenes to deliver its product candidates, which may limit the safety, purity, and efficacy or potency of such product candidates.

Neurogene’s current approach is to identify, develop and commercialize gene therapy product candidates using an AAV9 capsid for delivery of therapeutic transgenes to certain kinds of cells.

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Although AAV9 has been tested in numerous clinical trials and is an approved serotype for one gene therapy product, Neurogene cannot be certain that its AAV9 product candidates will successfully advance through preclinical studies and clinical trials, or that they will not cause significant adverse events or toxicities. Neurogene also cannot be certain that it will be able to avoid triggering toxicities in its future preclinical studies or clinical trials or that its chosen routes of administration to deliver such therapies will not cause unforeseen side effects or other challenges. Although AAV9 has been shown to facilitate biodistribution and cell transduction to the central nervous system (“CNS”), the potentially limited levels of AAV9 transduction of cells in the CNS and certain retinal cells may limit the potential efficacy or potency of any of Neurogene’s product candidates, including NGN-401 and NGN-101.

Neurogene intends to identify and develop novel gene therapy product candidates, which makes it difficult to predict the time, cost and potential success of product candidate development.

A key part of Neurogene’s business strategy is to identify and develop additional product candidates. As such, Neurogene’s future success depends on the successful development of novel therapeutic approaches, including by utilizing its EXACT technology or other gene regulation technology. Neurogene’s preclinical research and clinical trials may initially show promise in identifying potential product candidates, yet fail to yield product candidates for a number of reasons. For example, although EXACT is designed to deliver therapeutic levels of transgene while avoiding off-target effects, there can be no assurance that any EXACT gene regulation will result in product candidates that are shown in clinical trials to be safe, pure and effective or potent.

To date, very few products that utilize gene transfer have been approved in the United States, Europe or other markets, and no products have been approved using Neurogene’s Expression Attenuation via Construct Timing (“EXACT”) technology. There have been a limited number of clinical trials of gene transfer technologies, with only very few product candidates ever approved by the FDA or comparable foreign regulatory authorities.

As a result, it is difficult for Neurogene to predict the time and cost of product candidate development, and Neurogene cannot predict whether the application of its approach to gene therapy will result in the identification, development, and regulatory approval of any product candidates, or that other gene therapy programs will not be considered better or more attractive. There can be no assurance that any development problems Neurogene experiences in the future related to its current gene therapy approaches or product candidates or any of its research programs will not cause significant delays or unanticipated costs, or that such development problems can be solved. Research programs to identify new product candidates require substantial technical, financial, and human resources. If Neurogene is unable to identify suitable gene therapy product candidates for preclinical and clinical development, Neurogene may not be able to successfully implement its business strategy, and may have to delay, reduce the scope of, suspend or eliminate one or more of its product candidates, clinical trials or future commercialization efforts, which would negatively impact its financial condition.

The disorders Neurogene seeks to treat have low prevalence and it may be difficult to identify and enroll patients with these disorders. If Neurogene experiences delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, its receipt of necessary regulatory approvals could be delayed or prevented.

Successful and timely completion of clinical trials will require that Neurogene enroll and maintain a sufficient number of patients. Patient enrollment is affected by many factors, including the size and nature of the patient population and competition for patients with other trials. Genetic diseases generally, and especially the rare diseases for which some of Neurogene’s current product candidates are targeted, have low incidence and prevalence. For example, Neurogene estimates global incidence of all 13 subtypes of Batten disease is approximately one in 100,000 live births, and the CLN5 Batten disease incidence, which is included in this estimate, is estimated to be even lower. Accordingly, it may be difficult for Neurogene to identify and timely recruit a sufficient number of eligible patients to conduct its clinical trials. Further, any natural history studies

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that Neurogene or its collaborators may conduct may fail to provide Neurogene with patients for its clinical trials because patients enrolled in the natural history studies may not be good candidates for Neurogene’s clinical trials, or may choose to not enroll in Neurogene’s clinical trials.

Trials may be subject to delays as a result of patient enrollment taking longer than anticipated or patient withdrawal. Neurogene may not be able to initiate or continue clinical trials for its product candidates if it is unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA, the European Medicines Agency (“EMA”) or other foreign regulatory authorities. Neurogene cannot predict how successful it will be at enrolling subjects in future clinical trials. Subject enrollment is affected by other factors including:

Even if Neurogene is able to enroll a sufficient number of patients in its clinical trials, it may have difficulty maintaining enrollment of such patients. Neurogene’s inability to enroll or maintain a sufficient number of patients would result in significant delays in completing clinical trials or receipt of marketing approvals and increased development costs, or may require Neurogene to abandon one or more clinical trials altogether.

Neurogene is substantially dependent on the success of its most advanced product candidates, NGN-401 and NGN-101, and its ongoing and anticipated clinical trials of such candidates may not be successful.

Neurogene’s future success is substantially dependent on its ability to timely obtain marketing approval for, and then successfully commercialize, its most advanced product candidates, NGN-401 and NGN-101. Neurogene is investing a majority of its efforts and financial resources into the research and development of these candidates. Neurogene is conducting a Phase 1/2 clinical trial of NGN-401 in patients with Rett syndrome and a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease. If topline results from its Phase 1/2 clinical trial of NGN-401 are successful, Neurogene anticipates initiating a pivotal clinical trial, pending future regulatory feedback on various aspects of development such as the pivotal trial design and manufacturing related requirements. If topline results from its Phase 1/2 clinical trial of NGN-101 are successful, Neurogene anticipates initiating a pivotal clinical trial or expanding the current Phase 1/2 clinical trial, pending future regulatory feedback on various aspects of development, such as the Phase 3 clinical trial design and manufacturing related requirements.

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NGN-401 and NGN-101 will require additional clinical development, evaluation of clinical, preclinical and manufacturing activities, marketing approval in multiple jurisdictions, substantial investment and significant marketing efforts before Neurogene generates revenues from product sales, if any. Neurogene is not permitted to market or promote these product candidates, or any other product candidates, before it receives marketing approval from the FDA and/or comparable foreign regulatory authorities, and Neurogene may never receive such marketing approvals.

The success of NGN-401 and NGN-101 will depend on a variety of factors. Neurogene does not have complete control over many of these factors, including certain aspects of clinical development and the regulatory submission process, potential threats to its intellectual property rights and the manufacturing, marketing, distribution and sales efforts of any future collaborator. Accordingly, Neurogene cannot guarantee that it will ever be able to generate revenue through the sale of these candidates, even if approved. If Neurogene is not successful in commercializing NGN-401 or NGN-101, or is significantly delayed in doing so, its business will be materially harmed.

Neurogene’s programs are focused on the development of therapeutics for patients with neurological diseases, which is a rapidly evolving area of science, and the approach Neurogene is taking to discover and develop product candidates is novel and may never lead to approved or marketable products.

The discovery and development of therapeutics for patients with neurological diseases is an emerging field, and the scientific discoveries that form the basis for Neurogene’s efforts to discover and develop product candidates are relatively new. The scientific evidence to support the feasibility of developing product candidates based on these discoveries is both preliminary and limited. Although Neurogene believes, based on its preclinical work, that its programs have the potential to be disease-modifying therapies, clinical results may not confirm this hypothesis or may only confirm it for certain alterations or certain indications. The patient populations for Neurogene’s product candidates are limited to those with specific neurological diseases. Neurogene cannot be certain that the patient populations for each specific disease will be large enough to allow Neurogene to successfully obtain approval and commercialize its product candidates and achieve profitability. Further, both Neurogene’s Phase 1/2 clinical trial of NGN-401 and Phase 1/2 clinical trial of NGN-101 will involve a small patient population. Because of the small sample sizes, the results of these trials may not be indicative of results of future clinical trials.

If Neurogene does not achieve its projected development goals in the time frames Neurogene announces and expects, the commercialization of NGN-401 or NGN-101 or any other product candidates may be delayed and, as a result, its stock price may decline.

From time to time, Neurogene estimates the timing of the anticipated accomplishment of various scientific, clinical, regulatory and other product development goals, which Neurogene sometimes refer to as milestones. These milestones may include the commencement or completion of scientific studies and clinical trials and the submission of regulatory filings. From time to time, Neurogene may publicly announce the expected timing of some of these milestones. All of these milestones are and will be based on numerous assumptions. The actual timing of these milestones can vary dramatically compared to its estimates, in some cases for reasons beyond its control. If Neurogene does not meet these milestones as publicly announced, or at all, the commercialization of NGN-401 or NGN-101 or any other product candidates may be delayed or never achieved and, as a result, its stock price may decline.

Preclinical and clinical development involves a lengthy and expensive process that is subject to delays and with uncertain outcomes, and results of earlier studies and trials may not be predictive of future clinical trial results. If Neurogene’s preclinical studies and clinical trials are not sufficient to support regulatory approval of any of its product candidates, Neurogene may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development of such product candidate.

Before obtaining marketing approval from regulatory authorities for the sale of any product candidate, Neurogene must complete preclinical studies, which are a lengthy, time consuming and expensive process with risk of high failure. The length of time of such testing may vary substantially according to the type, complexity

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and novelty of the program, and often can be several years or more per program. Delays associated with programs for which Neurogene is conducting preclinical testing and studies may cause us to incur additional operating expenses. However, after conducting preclinical studies, Neurogene must then conduct extensive clinical trials to demonstrate the safety, purity, and efficacy or potency of its product candidate in humans. Neurogene’s clinical trials may not be conducted as planned or completed on schedule, if at all. For example, Neurogene depends on the availability of NHPs to conduct certain preclinical studies that Neurogene is required to complete prior to submitting an IND and initiating clinical development. There is currently a global shortage of NHPs available for biological product development. This could cause the cost of obtaining NHPs for its future preclinical studies to increase significantly and, if the shortage continues, could also result in delays to Neurogene’s development timelines.

Furthermore, failure can occur at any time during the preclinical study or clinical trial process, and the outcome of preclinical studies and early-stage clinical trials may not be predictive of the success of later clinical trials, especially as Neurogene’s initial clinical trials do not contain a control arm. In addition, Neurogene has designed its initial clinical trials with relatively small cohorts before expanding in size and dosing in subsequent cohorts. If safety issues arise in an early cohort, Neurogene may be delayed or prevented from dose escalating or subsequently expanding into larger trial cohorts.

Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of their product candidates. Earlier gene therapy clinical trials conducted by others also utilized AAV vectors. However, these studies should not be relied upon as evidence that Neurogene’s planned clinical trials will succeed. In addition, Neurogene expects to rely on patients to provide feedback on measures, which are subjective and inherently difficult to evaluate. These measures can be influenced by factors outside of its control, and can vary widely from day to day for a particular patient, and from patient to patient and from site to site within a clinical trial.

Neurogene cannot be sure that the FDA or comparable foreign regulatory authorities will agree with its clinical development plan. Neurogene is conducting a Phase 1/2 clinical trial of NGN-401 in patients with Rett syndrome and a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease. If the FDA or comparable regulatory authorities requires Neurogene to conduct additional trials or enroll additional patients, its development timelines may be delayed. Neurogene cannot be sure that submission of an IND application, clinical trial application (“CTA”) or similar application will result in the FDA or comparable foreign regulatory authorities, as applicable, allowing clinical trials to begin in a timely manner, if at all. Moreover, even if these trials begin, issues may arise that could cause regulatory authorities to require Neurogene to suspend or terminate such clinical trials. Events that may prevent successful or timely initiation or completion of clinical trials include: inability to generate sufficient preclinical, toxicology or other in vivo or in vitro data to support the initiation or continuation of clinical trials; delays in reaching a consensus with regulatory authorities on study design or implementation of the clinical trials; delays or failure in obtaining regulatory authorization to commence a trial; delays in reaching agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites; delays in identifying, recruiting and training suitable clinical investigators; delays in obtaining required IRB approval at each clinical trial site; difficulties in patient enrollment in Neurogene’s clinical trials for a variety of reasons; delays in manufacturing, testing, releasing, validating or importing/exporting sufficient stable quantities of its product candidates for use in clinical trials or the inability to do any of the foregoing; failure by its CROs, other third parties or Neurogene to adhere to clinical trial protocols; failure to perform in accordance with the FDA’s or any other regulatory authority’s good clinical practices (“GCPs”) or applicable regulatory guidelines in other countries; changes to the clinical trial protocols; clinical sites deviating from trial protocol or dropping out of a trial; changes in regulatory requirements and guidance that require amending or submitting new clinical protocols; selection of clinical endpoints that require prolonged periods of observation or analyses of resulting data; transfer of manufacturing processes to larger-scale facilities operated by a CDMO and delays or

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failure by its CDMOs or Neurogene to make any necessary changes to such manufacturing process; and third parties being unwilling or unable to satisfy their contractual obligations to Neurogene.

Neurogene could also encounter delays if a clinical trial is placed on clinical hold, suspended or terminated by Neurogene, the IRBs of the institutions in which such trials are being conducted, or the FDA, the competent authorities and/or ethics committees of the UK, Australia, EU Member States or other regulatory authorities, if a clinical trial is recommended for suspension or termination by the DSMB or equivalent body for such trial, or on account of changes to federal, state, or local laws. If Neurogene is required to conduct additional clinical trials or other testing of NGN-401 or NGN-101 or any other product candidates beyond those that Neurogene contemplates, if Neurogene is unable to successfully complete clinical trials of NGN-401 or NGN-101 or any other product candidates, if the results of these trials are not positive or are only moderately positive or if there are safety concerns, its business and results of operations may be adversely affected and Neurogene may incur significant additional costs.

In addition, even if Neurogene is able to successfully complete clinical trials for NGN-401 or NGN-101, it cannot guarantee that the FDA or foreign regulatory authorities will interpret the results as Neurogene does, and more trials could be required before Neurogene submits its product candidates for approval. This is particularly true for clinical trials in very rare diseases, such as with Neurogene’s Phase 1/2 clinical trial of NGN-101 for the treatment of CLN5 Batten disease and Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome, where the very small patient population makes it difficult to conduct two traditional, adequate and well-controlled studies. In such cases, the FDA or comparable foreign regulatory authorities are often required or permitted to exercise flexibility in approving therapies for such diseases, but obtaining flexibility is uncertain and may never occur. Moreover, results acceptable to support approval in one jurisdiction may be deemed inadequate by another regulatory authority to support regulatory approval in the other jurisdiction. To the extent that the results of the trials are not satisfactory to the FDA or applicable regulatory authorities for support of a marketing application, Neurogene may be required to expend significant resources, which may not be available to it, to conduct additional trials in support of potential approval of its product candidates.

Preliminary, “topline” or interim data from Neurogene’s clinical trials that it announces or publishes from time to time may change as more patient data becomes available and are subject to audit and verification procedures.

From time to time, Neurogene may publicly disclose preliminary, interim or topline data from its preclinical studies and clinical trials, which are based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data. Neurogene also makes assumptions, estimations, calculations and conclusions as part of its analyses of these data without the opportunity to fully and carefully evaluate complete data. Preliminary, interim, or topline results also remain subject to audit and verification procedures that may result in the final data being materially different from the preliminary data previously disclosed. These preliminary, interim or topline data are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data become available or as patients from its clinical trials continue other treatments. As a result, preliminary, interim and topline data should be viewed with caution until final data are available. Further, others, including regulatory agencies, may not accept or agree with its assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the value of the particular product candidate, the approvability or commercialization of a particular product candidate and Neurogene’s company in general. In addition, the information Neurogene chooses to publicly disclose regarding a particular preclinical study or clinical trial is based on what is typically extensive information, and you or others may not agree with what Neurogene determines is material or otherwise appropriate information to include in its disclosure. If the preliminary, interim or topline data that Neurogene reports differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, Neurogene’s ability to obtain approval for, and commercialize, NGN-401 or NGN-101 or any other product candidate may be harmed, which could harm its business, operating results, prospects or financial condition. In addition,

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differences between preliminary, interim or topline data and final data could significantly harm Neurogene’s business prospects and may cause the trading price of its common stock to fluctuate significantly.

Neurogene’s current or future clinical trials may reveal significant adverse events or undesirable side effects not seen in its preclinical studies and may result in a safety profile that could halt clinical development, inhibit regulatory approval or limit commercial potential or market acceptance of any of NGN-401 or NGN-101 or any other product candidates or result in potential product liability claims.

Results of Neurogene’s clinical trials could reveal a high and unacceptable severity and prevalence of side effects, adverse events or unexpected characteristics. While its Phase 1/2 clinical trials have not shown any such characteristics to date, Neurogene has not yet completed the clinical trials. If significant adverse events or other side effects are observed in any of its current or future clinical trials, Neurogene may have difficulty recruiting patients to such trials, patients may drop out of its trials, patients may be harmed, or Neurogene may be required to abandon the trials or its development efforts of one or more product candidates altogether, including NGN-401 or NGN-101. Neurogene, the FDA, EMA, or other applicable regulatory authorities, or an IRB, may require suspension of any clinical trials of NGN-401 or NGN-101 or any other product candidates at any time for various reasons, including a finding that subjects or patients in such trials are being exposed to unacceptable health risks or adverse side effects. Some potential products developed in the biotechnology industry that initially showed therapeutic promise in early-stage trials have later been found to cause side effects that prevented their further development. Even if the side effects do not preclude a product candidate from obtaining or maintaining marketing approval, undesirable side effects may inhibit market acceptance of an approved product due to its tolerability versus other therapies. In addition, as gene replacement has a potentially life-long activity, with no ability to withdraw the product as with other treatment modalities, this profile could prolong the duration of undesirable side effects, which could also inhibit market acceptance. Treatment-emergent adverse events could also affect patient recruitment or the ability of enrolled subjects to complete its clinical trials or could result in potential product liability claims. Potential side effects associated with NGN-401 or NGN-101 or any other product candidates may not be appropriately recognized or managed by the treating medical staff, as toxicities resulting from NGN-401 or NGN-101 or any other product candidates may not be normally encountered in the general patient population and by medical personnel. Any of these occurrences could harm Neurogene’s business, financial condition, results of operations and prospects significantly.

In addition, even if Neurogene successfully advances NGN-401 or NGN-101 or any other product candidates through clinical trials, such trials will only include a limited number of patients and limited duration of follow up to such product candidates. As a result, Neurogene cannot be assured that adverse effects of NGN-401 or NGN-101 or any other product candidates will not be uncovered when a significantly larger number of patients are exposed to such product candidate after approval, or a significantly longer follow up post-dosing is obtained as part of regulators’ recommendations for long-term follow up of clinical study subjects treated with gene therapy. Further, any clinical trials may not be sufficient to determine the effect and safety consequences of using Neurogene’s product candidates over a multi-year period.

Neurogene has expended substantial efforts and costs testing its EXACT technology in preclinical studies of NGN-401, including completing toxicology studies prior to the FDA providing clearance of the IND for NGN-401. Neurogene, however, cannot guarantee that significant adverse effects will not be seen in clinical trials for NGN-401, which could result in clinical holds, delays, suspension or withdrawal of Neurogene’s IND. If any of the foregoing events occur or if NGN-401 or NGN-101 or any other product candidates prove to be unsafe, Neurogene’s entire pipeline could be affected, which would have a material adverse effect on its business, financial condition, results of operations and prospects.

Neurogene may expend its limited resources to pursue a particular product candidate, such as NGN-401 or NGN-101, and fail to capitalize on candidates that may be more profitable or for which there is a greater likelihood of success.

Because Neurogene has limited financial and managerial resources, Neurogene intends to focus its research and development efforts on certain selected product candidates. For example, Neurogene is initially allocating

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significant resources to its most advanced product candidates, NGN-401 and NGN-101. As a result, Neurogene may forgo or delay pursuit of opportunities with other potential candidates that may later prove to have greater commercial potential. Neurogene’s resource allocation decisions may cause Neurogene to fail to capitalize on viable commercial products or profitable market opportunities. Neurogene’s spending on current and future research and development programs for specific indications may not yield any commercially viable product candidates. If Neurogene does not accurately evaluate the commercial potential or target market for a particular product candidate, it may relinquish valuable rights to that candidate through collaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for Neurogene to retain sole development and commercialization rights to such candidate.

Even if regulatory approval is obtained, any approved products resulting from NGN-401 or NGN-101 or any other product candidate may not achieve adequate market acceptance among clinicians, patients, healthcare third-party payors and others in the medical community necessary for commercial success and Neurogene may not generate any future revenue from the sale or licensing of such products.

Even if regulatory approval is obtained for NGN-401 or NGN-101 or any other product candidates, Neurogene’s product candidates may not gain market acceptance among physicians, patients, healthcare payors or the medical community. Neurogene may not generate or sustain revenue from sales of the product due to factors such as whether the product can be sold at a competitive cost and whether it will otherwise be accepted in the market. There is currently one FDA-approved product and multiple other product candidates in various stages of development for the treatment of Rett syndrome. Market participants with significant influence over acceptance of new treatments, such as clinicians and third-party payors, may not adopt a gene therapy replacement with a target product profile such as that of NGN-401 or NGN-101 or for their targeted indications, and Neurogene may not be able to convince the medical community and third-party payors to accept and use, or to provide favorable reimbursement for, any product candidates developed by Neurogene or its existing or future collaborators. Market acceptance of NGN-401 or NGN-101 or any other product candidates will depend on many factors, including factors that are not within Neurogene’s control.

Sales of biological products also depend on the willingness of clinicians to prescribe the treatment. Neurogene cannot predict whether clinicians, clinicians’ organizations, hospitals, other healthcare providers, government agencies or private insurers will determine that any of its approved products are safe, therapeutically effective or potent, cost effective or less burdensome as compared with competing treatments. If NGN-401 or NGN-101 or any other product candidate is approved but does not achieve an adequate level of acceptance by such parties, Neurogene may not generate or derive sufficient revenue from that product and may not become or remain profitable.

Neurogene has never commercialized a product candidate and may lack the necessary expertise, personnel and resources to successfully commercialize a product candidate on its own or together with suitable collaborators.

Neurogene has never commercialized a product candidate, and it currently has no sales force, marketing or distribution capabilities. To achieve commercial success for a product candidate, which Neurogene may license to others, Neurogene may rely on the assistance and guidance of those collaborators. For a product candidate for which Neurogene retains commercialization rights and marketing approval, Neurogene will have to develop its own sales, marketing and supply organization or outsource these activities to a third party. Factors that may affect its ability to commercialize a product candidate, if approved, on its own include recruiting and retaining adequate numbers of effective sales and marketing personnel, developing adequate educational and marketing programs to increase public acceptance of its approved product candidate, ensuring regulatory compliance of Neurogene’s company, employees and third parties under applicable healthcare laws and other unforeseen costs associated with creating an independent sales and marketing organization. Developing a sales and marketing organization will be expensive and time-consuming and could delay the launch of a product candidate upon approval. Neurogene may not be able to build an effective sales and marketing organization. If Neurogene is

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unable to build its own distribution and marketing capabilities or to find suitable partners for the commercialization of an approved product candidate, Neurogene may not generate revenues from them or be able to reach or sustain profitability.

Neurogene has never completed any late-stage clinical trials and it may not be able to file an IND application or other applications for regulatory approval to commence additional clinical trials on the timelines it expects. Even if Neurogene is able to complete such trials, the FDA or comparable foreign regulatory authorities may not permit Neurogene to proceed or could suspend or terminate any such trial after it has been initiated.

Neurogene is early in its development efforts and will need to successfully complete later-stage and pivotal clinical trials in order to obtain FDA or comparable foreign regulatory approval to market its product candidates. Carrying out clinical trials and the submission of a successful IND or CTA is a complicated process. Neurogene has not yet completed a Phase 1/2 clinical trial and has limited experience as a company in preparing, submitting and prosecuting regulatory filings. If topline results from its Phase 1/2 clinical trial of NGN-401 are successful, Neurogene intends to engage with the FDA and other comparable foreign regulators to determine the requirements to support initiation of a pivotal clinical trial. If topline results from its Phase 1/2 clinical trial of NGN-101 are successful, Neurogene intends to engage with the FDA and other comparable foreign regulators to determine if there is a streamlined pathway to approval for NGN-101 for the treatment of CLN5 Batten disease. However, regulatory authorities may recommend changes to the study designs for NGN-401 or NGN-101, including the number and size of registrational clinical trials required to be conducted in such programs. In addition, regulatory authorities could require manufacturing changes or have Neurogene implement additional analytical processes prior to initiation of a future clinical trial. Consequently, Neurogene may be unable to successfully and efficiently execute and complete necessary clinical trials in a way that leads to regulatory submission and approval of its product candidates. Additionally, even if regulatory authorities agree with the design and implementation of the clinical trials set forth in a regulatory meeting, such regulatory authorities may change their requirements in the future. The FDA or comparable foreign regulatory authorities may require the analysis of data from trials assessing different doses of the product candidate alone or in combination with other therapies to justify the selected dose prior to the initiation of large trials in a specific indication. Any delays or failure to initiate clinical trials, or obtain regulatory approvals for its trials may prevent Neurogene from completing its clinical trials or commercializing its products on a timely basis, if at all. Neurogene is subject to similar risks related to the review and authorization of its protocols and amendments by comparable foreign regulatory authorities.

For its preclinical pipeline, if the IND-enabling studies support a decision to advance into clinical development, Neurogene would plan to submit an IND or CTA with a foreign regulatory authority. Neurogene may not be able to file the IND or CTA in accordance with its desired timelines for future product candidates. For example, Neurogene may experience manufacturing delays or other delays with IND-enabling studies, including with suppliers, study sites, or third-party contractors and vendors on which Neurogene depends. Moreover, Neurogene cannot be sure that submission of an IND application will result in the FDA or comparable foreign regulatory authorities allowing further clinical trials to begin, or that, once begun, issues will not arise that lead Neurogene to suspend or terminate such clinical trials.

Risks Related to Manufacturing

Gene therapies are novel, complex and difficult to manufacture. Neurogene could experience manufacturing problems that result in delays in the development or commercialization of its product candidates or otherwise harm its business.

The manufacture of gene therapy products is technically complex and necessitates substantial expertise and capital investment. Production difficulties caused by unforeseen events may delay the availability of material for Neurogene’s clinical studies. While Neurogene is currently establishing its own manufacturing facility to provide clinical and commercial supply of its product candidates, Neurogene expects to rely on contract manufacturers

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for certain portions of its manufacturing needs for the foreseeable future, such as those related to research grade material for its early preclinical studies. Neurogene has also relied on a third-party contract manufacturer to manufacture clinical supply for its Phase 1/2 clinical trial of NGN-101.

The manufacturers of biological and pharmaceutical products must comply with strictly enforced cGMP requirements, state and federal regulations, as well as foreign requirements when applicable. Any failure of Neurogene or its CDMOs to adhere to or document compliance to such regulatory requirements could lead to a delay or interruption in the availability of Neurogene’s program materials for clinical trials or enforcement action from the FDA, EMA or other foreign regulatory authorities. If Neurogene or its manufacturers were to fail to comply with the FDA, EMA or other regulatory authority, it could result in sanctions being imposed on Neurogene, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or products, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of Neurogene’s product candidates. Neurogene’s potential future dependence upon others for the manufacture of its product candidates may also adversely affect Neurogene’s future profit margins and its ability to commercialize any product candidates that receive regulatory approval on a timely and competitive basis.

Biological products are inherently difficult to manufacture. Although Neurogene believes that the manufacture of its product candidates may be simplified due to their shared raw materials and other similarities, Neurogene cannot be certain that this will be the case and it may be required to develop manufacturing methods that ultimately differ significantly between product candidates, which would require that Neurogene invest substantial time and capital to develop suitable manufacturing methods. Neurogene’s program materials are manufactured using technically complex processes requiring specialized equipment and facilities, highly specific raw materials, cells, and reagents, and other production constraints. Neurogene’s production process requires a number of highly specific raw materials, cells and reagents with limited suppliers. Even though Neurogene aims to have backup supplies of raw materials, cells and reagents whenever possible, Neurogene cannot be certain they will be sufficient if Neurogene’s primary sources are unavailable. A shortage of a critical raw material, cell line, or reagent, or a technical issue during manufacturing may lead to delays in clinical development or commercialization plans. Neurogene is particularly susceptible to any shortages, delays or its inability to obtain suitable raw materials, given that all of Neurogene’s current and planned product candidates require this starting material. Any changes in the manufacturing of components of the raw materials Neurogene uses could result in unanticipated or unfavorable effects in Neurogene’s manufacturing processes, resulting in delays.

Once the biological products are manufactured, the product must be analyzed utilizing assays and meet pre-determined specifications in order to be used in certain preclinical studies, in any clinical trial, and if approval is obtained, for commercial distribution. This testing is performed in-house and at third-party contract manufacturers. Delays or other unexpected obstacles in performing the tests and obtaining the results in-house or at a third-party contractor could result in unanticipated impact to Neurogene’s ability to supply material as needed for pre-clinical, clinical, or commercial needs.

Neurogene and its contract manufacturers for AAV9 are subject to significant regulation with respect to manufacturing of Neurogene’s products. The third-party manufacturing facilities on which Neurogene relies, Neurogene’s in-house manufacturing facility, and any manufacturing facility that Neurogene may have in the future, may have limited capacity or fail to meet the applicable stringent regulatory requirements.

Neurogene currently has relationships with a limited number of suppliers for the raw materials, including plasmids and virus banks, required by the manufacturing processes of Neurogene’s product candidates. Virus intended for use in its early preclinical studies has been and can be externally supplied; however, if Neurogene experiences slowdowns or problems with its in-house manufacturing facility and is unable to establish or scale its internal manufacturing capabilities, Neurogene will need to continue to contract with manufacturers to produce the preclinical, clinical and commercial supply and such supply will be more uncertain and subject to delays. In addition, each supplier may require licenses to manufacture certain components of the supply if such processes

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are not owned by the supplier or in the public domain and Neurogene may be unable to license such intellectual property rights on reasonable commercial terms or to transfer or sublicense the intellectual property rights it may have with respect to such activities.

All entities involved in the preparation of therapeutics for clinical trials or commercial sale, including Neurogene’s existing contract manufacturers for components of its product candidates, are subject to extensive regulation. Components of a finished therapeutic product approved for commercial sale or used in late-stage clinical trials must be manufactured in accordance with cGMP. These regulations govern manufacturing processes and procedures (including recordkeeping) and the implementation and operation of quality systems to control and assure the quality of investigational products and products approved for sale. Poor control of production processes can lead to the introduction of adventitious agents or other contaminants, or to inadvertent changes in the properties or stability of Neurogene’s product candidates that may not be detectable in final product testing. Neurogene or its contract manufacturers must supply all necessary documentation in support of a biologics license application (“BLA”) or marketing authorization application (“MAA”) on a timely basis. Neurogene’s facilities and quality systems and the facilities and quality systems of some or all of its third-party contractors must pass a pre-approval inspection for compliance with the applicable regulations as a condition of regulatory approval of Neurogene’s current or future product candidates. In addition, regulatory authorities may, at any time, audit or inspect a manufacturing facility involved with the preparation of Neurogene’s current or future product candidates or the associated quality systems for compliance with the regulations applicable to the activities being conducted, and they could put a hold on one or more of Neurogene’s clinical trials if the facilities of Neurogene’s CDMOs do not pass such audit or inspections. If these facilities do not pass a pre-approval plant inspection, the FDA or other foreign regulatory agency approval of the products will not be granted.

Regulatory authorities also may, at any time following approval of a product for sale, inspect or audit Neurogene’s manufacturing facilities or those of its third-party contractors. If any such inspection or audit identifies a failure to comply with applicable regulations or if a violation of Neurogene’s product specifications or applicable regulations occurs independent of such an inspection or audit, Neurogene or the relevant regulatory authority may require remedial measures that may be costly and/or time-consuming for Neurogene or a third party to implement, and that may include the temporary or permanent suspension of a clinical trial or commercial sales or the temporary or permanent closure of a facility. Any such remedial measures imposed upon Neurogene or third parties with whom it contracts could harm Neurogene’s business. If Neurogene or any of its third-party manufacturers fail to maintain regulatory compliance, the FDA or other foreign regulatory agencies can impose regulatory sanctions including, among other things, refusal to approve a pending application for a new drug product or biologic product, or revocation of a pre-existing approval. As a result, Neurogene’s business, financial condition and results of operations may be harmed.

Additionally, if supply from one approved manufacturer is interrupted, there could be a significant disruption in commercial supply. An alternative manufacturer would need to be qualified through a BLA and/or MAA supplement, which could result in further delay. The regulatory agencies may also require additional studies if a new manufacturer is relied upon for commercial production. Switching manufacturers may involve substantial costs and is likely to result in a delay in Neurogene’s desired clinical and commercial timelines.

These factors could cause the delay of clinical trials, regulatory submissions, required approvals or commercialization of Neurogene’s product candidates, cause Neurogene to incur higher costs and prevent it from commercializing its products successfully, if approved. Further, if Neurogene’s suppliers fail to meet contractual requirements, and Neurogene is unable to secure one or more replacement suppliers capable of production at a substantially equivalent cost, Neurogene’s clinical trials may be delayed or Neurogene could lose future potential revenue, if any.

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Neurogene depends on third-party suppliers for materials used in the manufacture of its product candidates, and the loss of these third-party suppliers or their inability to supply Neurogene with adequate materials could harm Neurogene’s business.

Neurogene relies on third-party suppliers for certain materials and components required for the production of its product candidates. Neurogene’s dependence on these third-party suppliers and the challenges it may face in obtaining adequate supplies of materials involve several risks, including limited control over pricing, availability and quality of supplies and delivery schedules. There is substantial demand and limited supply for certain of the raw materials used to manufacture gene therapy products. As a small company, Neurogene’s negotiation leverage is limited and it is likely to get lower priority than its competitors that are larger than Neurogene. Neurogene cannot be certain that its suppliers will continue to provide it with the quantities of raw materials that Neurogene requires or satisfy Neurogene’s anticipated specifications and quality requirements. Any supply interruption in limited or sole sourced raw materials could materially harm Neurogene’s ability to manufacture its product candidates until a new source of supply, if any, could be identified and qualified. Neurogene may be unable to find a sufficient alternative supply channel in a reasonable time or on commercially reasonable terms. Any performance failure on the part of Neurogene’s suppliers could delay the development and potential commercialization of Neurogene’s product candidates, including limiting supplies necessary for clinical trials and regulatory approvals, which would have a material adverse effect on Neurogene’s business.

Delays in developing Neurogene’s manufacturing capabilities or failure to achieve operating efficiencies from such capabilities may require Neurogene to devote additional resources and management time to manufacturing operations and may delay Neurogene’s product development timelines.

Neurogene has a GMP manufacturing facility located in Houston, Texas that includes process, analytical and bioanalytical development labs with experienced teams. NGN-401 was manufactured at Neurogene’s Houston facility and clinical-grade product is available for dosing in the Phase 1/2 clinical trial of NGN-401 that is currently enrolling patients. However, Neurogene will need to conduct additional NGN-401 manufacturing campaigns to generate additional clinical supply, as well as supply for its preclinical studies for its discovery programs, and Neurogene may not be able to satisfy such supply through production at its own facility.

Other risks relating to the manufacture of biologics and drug products include: production interruptions, delays in quality/release testing, equipment malfunctions, facility contamination, labor problems, natural disasters, disruption in utility services, terrorist activities, war, cases of force majeure, acts of god (such as public health crises) or other events beyond Neurogene’s control and, in each case, could result in delays in Neurogene’s production or difficulties in maintaining compliance with applicable regulatory requirements.

Any contamination or interruption in Neurogene’s manufacturing process, shortages of raw materials or failure of Neurogene’s suppliers to deliver necessary components could result in delays in Neurogene’s clinical development or marketing schedules.

Given the nature of gene therapy manufacturing, there is a risk of contamination. Any contamination could adversely affect Neurogene’s ability to produce product candidates on schedule and could, therefore, harm its results of operations and cause reputational damage. Some of the raw materials required in Neurogene’s manufacturing process are derived from biologic sources. Such raw materials are difficult to procure and may be subject to contamination or recall. A material shortage, contamination, recall or restriction on the use of biologically derived substances in the manufacture of Neurogene’s product candidates could adversely impact or disrupt the commercial manufacturing or the production of clinical material, which could adversely affect Neurogene’s development timelines and its business, financial condition, results of operations and prospects.

Neurogene may not be able to successfully manufacture its product candidates in sufficient quality and quantity, which would delay or prevent Neurogene from developing its product candidates and commercializing resulting approved products, if any.

To date, Neurogene has manufactured NGN-401 in quantities and quality adequate for preclinical, toxicology and clinical studies. In order to conduct clinical trials for a product candidate and for

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commercialization of the resulting product if that product candidate is approved for sale, Neurogene will need to manufacture product candidates in additional cGMP campaigns or in larger batch sizes. Neurogene may not be able to successfully repeat or increase the manufacturing capacity for any of its product candidates in a timely or cost-effective manner or at all. Significant changes or scale-up of manufacturing may require additional validation studies, which are costly and which regulatory authorities must review and approve. In addition, quality issues may arise during those changes or scale-up activities. If Neurogene is unable to successfully manufacture any of its product candidates in sufficient quality and quantity, the development of that product candidate and regulatory approval or commercial launch for any resulting products may be delayed or there may be a shortage in supply, which could significantly harm Neurogene’s business.

Changes in methods of product candidate manufacturing or formulation may result in additional costs or delay.

As product candidates proceed through preclinical studies to late-stage clinical trials towards potential approval and commercialization, it is common that various aspects of the development program, such as manufacturing methods and formulation, are altered along the way in an effort to optimize processes and product characteristics. Such changes carry the risk that they will not achieve Neurogene’s intended objectives. Any such changes could cause Neurogene’s product candidates to perform differently and affect the results of planned clinical trials or other future clinical trials conducted with the materials manufactured using altered processes. Such changes may also require additional testing, FDA notification or approval from the FDA or foreign regulatory agencies. This could delay completion of clinical trials, require the conduct of bridging clinical trials or the repetition of one or more clinical trials, increase clinical trial costs, delay approval of Neurogene’s product candidates and jeopardize Neurogene’s ability to commence sales and generate revenue. In addition, Neurogene may be required to make significant changes to its upstream and downstream processes across its pipeline, which could delay the development of Neurogene’s future product candidates.

Risks Related to Neurogene’s Reliance on Third Parties

Neurogene has a number of academic collaborations, and currently relies on its collaboration with the University of Edinburgh for certain aspects of its preclinical research and development programs, including working in collaboration to discover and preclinically develop its lead product candidate for Rett syndrome and its near-term future pipeline. Failure or delay of the University of Edinburgh or any other collaborator to fulfil all or part of its obligations under its agreement with Neurogene, a breakdown in collaboration between the parties or a complete or partial loss of the relationship would materially harm Neurogene’s business.

Neurogene’s discovery engine is supplemented by academic collaborations to expand its platform, which Neurogene relies upon to advance discovery and development of its product candidates. For example, Neurogene’s collaboration with the University of Edinburgh is critical to its business. In December 2020, Neurogene entered into a Master Collaboration Agreement (the “MCA”) with the University of Edinburgh, which Neurogene relies on to conduct certain aspects of the preclinical development of its pipeline candidates, including NGN-401 and all of Neurogene’s early-stage pipeline product candidates. Further, in March 2022, Neurogene entered into an exclusive license agreement with the University of Edinburgh for, with respect to certain University of Edinburgh-owned technology, a worldwide, exclusive, sublicensable license to develop, have developed, use, manufacture, have manufactured, supply, have supplied, sell, have sold, offer for sale, commercialize, import, export, register, reproduce, dispose of or otherwise exploit any products, processes, components, services and/or technologies incorporating the technology for the prevention or treatment of disease or medical or genetic conditions in humans. Neurogene also currently relies on the University of Edinburgh for portions of preclinical research capabilities under the direction of Dr. Stuart Cobb, Professor in Translational Neuroscience at the University of Edinburgh and Neurogene’s Chief Scientific Officer. Pursuant to the MCA, Neurogene and the University of Edinburgh agreed to collaborate on certain research and development projects (the “Projects”), and Neurogene agreed to provide funding for such Projects. In exchange for such funding, the University of Edinburgh grants Neurogene an option to exclusively license any intellectual property arising from

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such Projects. Either party has the right in certain circumstances to terminate the collaboration pursuant to the terms of the MCA. If the MCA is not renewed or is terminated, Neurogene’s pipeline of product candidates would be significantly adversely affected and its business would be materially harmed.

The term of the research funding portion of the MCA, under which Neurogene has the ability to acquire exclusive rights to additional technology and gene therapy products, expires in March 2024. Neurogene is currently seeking an extension to the MCA for another three-year term, but Neurogene cannot guarantee that this extension will be made on equally favorable terms, if at all. Neurogene may have disagreements with the University of Edinburgh with respect to the interpretation of the MCA, use of resources or otherwise that could cause the parties’ relationship to deteriorate. As a result, the University of Edinburgh may reduce focus on, and resources allocated to, Neurogene’s programs, potentially delaying or terminating Neurogene’s ability to advance product candidates through preclinical studies. Additionally, if Dr. Cobb were to leave the University of Edinburgh or to otherwise no longer be meaningfully involved with Neurogene, Neurogene’s preclinical research and development capabilities may be substantially reduced.

Further, under the MCA, the University of Edinburgh is primarily responsible for prosecuting and maintaining Neurogene’s licensed intellectual property, and it may fail to properly prosecute, maintain or defend such intellectual property. In such event, if Neurogene is unable to otherwise maintain or defend such intellectual property, Neurogene could face the potential invalidation of the intellectual property or be subjected to litigation or arbitration, any of which would be time-consuming and expensive. To enforce the licensed intellectual property rights under the MCA, Neurogene will need to coordinate with the University of Edinburgh, which could slow down or hamper its ability to enforce its licensed intellectual property rights. If this happens, Neurogene could face increased competition that could materially and adversely affect its business. For a further description of the MCA, see “Neurogene’s Business—License Agreements.”

Additionally, in May 2019, Neurogene entered into an exclusive license agreement with the University of North Carolina (“UNC”) for, with respect to the UNC invention known as “Optimized CLN5 Genes and Expression Cassettes and Their Use,” a worldwide, exclusive, sublicensable license to make, use, sell, have made, have sold, offer for sale and import any method or process, composition, product, or component part thereof for the prevention or treatment of disease or medical or genetic conditions, including CLN5 Batten disease or other diseases stemming from dysfunction of the CLN5 gene.

Neurogene also currently has or may in the future engage in other academic collaborations to supplement its internal discovery and product development program. While these academic institutions have contractual obligations to Neurogene, they are independent entities and are not under Neurogene’s control or the control of Neurogene’s officers or directors. Neurogene’s research and licensing agreements with academic collaborators generally provide academic collaborators with license maintenance fees, development and regulatory milestone payments, royalties on net sales of products and a portion of sublicense income that Neurogene receives. Upon the scheduled expiration of any academic collaboration, Neurogene may not be able to renew the related agreement, or any renewal could be on terms less favorable to Neurogene than those contained in the existing agreement. Furthermore, either Neurogene or the academic institution generally may terminate the sponsored research agreement for convenience following a specified notice period. If any of these academic institutions decides to not renew or to terminate the related agreement or decides to devote fewer resources to such activities, Neurogene’s discovery efforts would be diminished, while its royalty obligations, if any, would continue unmodified.

Neurogene currently relies on and intends in the future to rely on third parties to conduct a significant portion of its preclinical studies and existing clinical trials and potential future clinical trials for product candidates, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.

Neurogene has engaged CROs or other third parties to conduct preclinical and IND enabling studies and its clinical trials, including its Phase 1/2 clinical trial of NGN-401 and Phase 1/2 clinical trial of NGN-101.

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Neurogene expects to continue to rely on third parties, including CROs, medical institutions and clinical investigators, to conduct those clinical trials. Any of these third parties may terminate their engagements with Neurogene, some in the event of an uncured material breach and some at any time for convenience. If any of Neurogene’s relationships with these third parties terminate, Neurogene may not be able to timely enter into arrangements with alternative third parties or do so on commercially reasonable terms, if at all. Switching or adding CROs involves substantial cost and requires management time and focus. In addition, there is a natural transition period when a new CRO commences work. As a result, delays occur, which can materially impact Neurogene’s ability to meet its desired clinical development timelines. Though Neurogene intends to carefully manage its relationships with CROs, there can be no assurance that Neurogene will not encounter challenges or delays in the future or that these delays or challenges will not have a material adverse impact on Neurogene’s business and financial condition.

In addition, any third parties conducting Neurogene’s clinical trials will not be Neurogene’s employees, and except for remedies available to Neurogene under its agreements with such third parties, Neurogene cannot control whether or not such third parties devote sufficient time and resources to Neurogene’s clinical programs. If these third parties do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to Neurogene’s clinical protocols, regulatory requirements or for other reasons, Neurogene’s clinical trials may be extended, delayed or terminated and it may not be able to obtain regulatory approval for or successfully commercialize its product candidates. Consequently, Neurogene’s results of operations and the commercial prospects for its product candidates would be harmed, its costs could increase substantially and its ability to generate revenue could be delayed significantly.

Further, while Neurogene’s reliance on these third parties for research and development activities will reduce Neurogene’s control over these activities, Neurogene will not be relieved of its responsibilities for ensuring that each of its studies and trials is conducted in accordance with the applicable protocol, legal, regulatory and scientific standards. For example, Neurogene will remain responsible for ensuring that each of its clinical trials is conducted in accordance with the general investigational plan and protocols for the trial. Moreover, the FDA requires Neurogene to comply with GCPs for conducting, recording and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity and confidentiality of trial participants are protected. Neurogene also is required to register ongoing clinical trials and post the results of completed clinical trials on a government-sponsored database, ClinicalTrials.gov, within specified timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions. If Neurogene or any of its CROs or other third parties, including trial sites, fail to comply with applicable GCPs, the clinical data generated in Neurogene’s clinical trials may be deemed unreliable and the FDA, EMA or comparable foreign regulatory authorities may require Neurogene to perform additional clinical trials before approving its marketing applications. Neurogene cannot assure you that upon inspection by a given regulatory authority, such regulatory authority will determine that any of Neurogene’s clinical trials complies with GCP regulations. In addition, Neurogene’s clinical trials must be conducted with product produced under cGMP conditions. Neurogene’s failure to comply with these regulations may require it to repeat clinical trials, which would delay the regulatory approval process.

In addition, principal investigators for Neurogene’s clinical trials may serve as scientific advisors or consultants to Neurogene from time to time and receive compensation in connection with such services. Under certain circumstances, Neurogene may be required to report some of these relationships to the FDA. The FDA may conclude that a financial relationship between Neurogene and a principal investigator has created a conflict of interest or otherwise affected interpretation of the trial. The FDA may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of Neurogene’s marketing applications by the FDA and may ultimately lead to the denial of marketing approval of NGN-401 and NGN-101 or any other product candidates.

Neurogene currently stores drug product for clinical trial sites in the United States, and currently relies on and expects in the future to rely on third parties to distribute product supplies for its clinical trials, as well as to

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store and distribute supply for clinical trial sites outside of the United States. Any performance failure on the part of Neurogene or Neurogene’s distributors could delay clinical development or marketing approval of its product candidates or commercialization of its products, if approved, producing additional losses and depriving Neurogene of potential revenue.

Risks Related to Neurogene’s Business and Operations

In order to successfully implement its plans and strategies, Neurogene will need to grow the size of its organization and it may experience difficulties in managing this growth.

Over time, Neurogene expects to experience significant growth in the number of its employees and the scope of its operations, particularly in the areas of preclinical and clinical biological product development, technical operations, clinical operations, regulatory affairs, manufacturing and, potentially, sales, marketing and distribution. To manage its anticipated future growth, Neurogene must continue to implement and improve its managerial, operational and financial personnel and systems, expand its facilities and continue to recruit and train additional qualified personnel. Due to its limited financial resources and the limited experience of its management team working together in managing a company with such anticipated growth, Neurogene may not be able to effectively manage the expansion of its operations or recruit and train additional qualified personnel. The expansion of Neurogene’s operations may lead to significant costs and may divert its management and business development resources. Any inability to manage growth could delay the execution of Neurogene’s business plans or disrupt its operations.

Neurogene is highly dependent on its key personnel and anticipates hiring new key personnel. If Neurogene is not successful in attracting and retaining highly qualified personnel, it may not be able to successfully implement its business strategy.

Neurogene’s ability to compete in the highly competitive biotechnology and pharmaceutical industries depends upon its ability to attract and retain highly qualified managerial, scientific and medical personnel. Neurogene is highly dependent on its managerial, scientific and medical personnel, including its Founder and Chief Executive Officer, President and Chief Financial Officer, and its Chief Scientific Officer and other key members of its leadership team. Neurogene’s executive officers may terminate their employment with Neurogene at any time. Neurogene does not maintain “key person” insurance for any of its executives or other employees. The loss of the services of its executive officers or other key employees could impede the achievement of its research, development and commercialization objectives and seriously harm its ability to successfully implement its business strategy. Furthermore, replacing executive officers and key personnel may be difficult and may take an extended period of time. If Neurogene does not succeed in attracting and retaining qualified personnel, it could materially and adversely affect its business, financial condition and results of operations. Neurogene could in the future have difficulty attracting and retaining experienced personnel and may be required to expend significant financial resources on its employee recruitment and retention efforts.

Neurogene’s future growth may depend, in part, on its ability to operate in foreign markets, where it would be subject to additional regulatory burdens and other risks and uncertainties.

Neurogene’s future growth may depend, in part, on its ability to develop and commercialize NGN-401 or NGN-101 or other product candidates in foreign markets for which Neurogene may rely on collaborations with third parties. Neurogene is not permitted to market or promote any product candidates before Neurogene receives regulatory approval from the applicable foreign regulatory authority, and may never receive such regulatory approval for any product candidates. To obtain separate regulatory approval in many other countries, Neurogene must comply with numerous and varying regulatory requirements of such countries regarding safety and efficacy and governing, among other things, clinical trials and commercial sales, pricing and distribution of NGN-401 or NGN-101 or other product candidates, and Neurogene cannot predict success in these jurisdictions. If Neurogene fails to comply with the regulatory requirements in international markets or to receive applicable marketing

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approvals, its target market will be reduced and its ability to realize the full market potential of NGN-401 or NGN-101 or other product candidates will be harmed and its business will be adversely affected. Moreover, even if Neurogene obtains approval of NGN-401 or NGN-101 or other product candidates and ultimately commercializes such product candidates in foreign markets, Neurogene would be subject to the risks and uncertainties, including the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements and reduced protection of intellectual property rights in some foreign countries.

Neurogene’s employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, CDMOs, suppliers and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.

Neurogene is exposed to the risk that its employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, CDMOs, suppliers and vendors acting for or on its behalf may engage in misconduct or other improper activities. It is not always possible to identify and deter misconduct by these parties and the precautions Neurogene takes to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting Neurogene from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.

Neurogene’s internal computer systems have suffered a security breach and in the future may, or those of any of its CROs, manufacturers, other contractors, third party service providers or consultants or potential future collaborators, may fail or suffer security or data privacy breaches or other unauthorized or improper access to, use of, or destruction of its proprietary or confidential data, employee data or personal data, which could result in additional costs, loss of revenue, significant liabilities, harm to its brand and material disruption of its operations.

Despite the implementation of security measures in an effort to protect systems that store its information, given their size and complexity and the increasing amounts of information maintained on its internal information technology systems and those of its third-party CROs, other contractors (including sites performing its clinical trials), third-party service providers and supply chain companies, consultants and other partners, these systems are potentially vulnerable to breakdown or other damage or interruption from service interruptions, system malfunction, natural disasters, terrorism, war and telecommunication and electrical failures, as well as security breaches from inadvertent or intentional actions by its employees, contractors, consultants, business partners and/or other third parties, or from cyber-attacks by malicious third parties, which may compromise its system infrastructure or lead to the loss, destruction, alteration or dissemination of, or damage to, its data. From time to time, Neurogene is subject to periodic business email compromise attack attempts. In August 2023, Neurogene discovered a business email compromise attack that resulted in the misappropriation of approximately $0.9 million. Neurogene cannot guarantee that the remedial measures it has implemented will prevent additional related, as well as unrelated incidents, or that it will be able to defend against or successfully remediate any such attacks that may occur in the future. If a material system failure, accident or security breach were to occur and cause interruptions in Neurogene’s operations or the operations of third-party collaborators, service providers, contractors and consultants, it could result in a material disruption of Neurogene’s development programs and significant reputational, financial, legal, regulatory, business or operational harm.

Further, since Neurogene sponsors clinical trials, any breach that compromises patient data and identities causing a breach of privacy could have significant adverse consequences on Neurogene’s business. For example, the loss of clinical trial data from completed or future clinical trials could affect trust in Neurogene to recruit for future clinical trials, result in delays in Neurogene’s regulatory approval efforts and significantly increase its costs to recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss, destruction, unavailability, alteration or dissemination of, or damage to, Neurogene’s data or applications, or inappropriate disclosure of confidential proprietary information, or for it to be believed or reported that any of these occurred, Neurogene could incur liability and reputational damage and the development and commercialization of NGN-101 or NGN-401 or other product candidates could be delayed.

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As Neurogene’s employees work remotely and utilize network connections, computers, and devices outside its premises or network, including working at home, while in transit and in public locations, there are risks to its information technology systems and data. Additionally, business transactions (such as acquisitions or integrations) could expose Neurogene to additional cybersecurity risks and vulnerabilities, as its systems could be negatively affected by vulnerabilities present in acquired or integrated entities’ systems and technologies.

While Neurogene has implemented security measures designed to protect against security incidents, there can be no assurance that these measures will be effective. Neurogene may be unable in the future to detect vulnerabilities in its information technology systems because such threats and techniques change frequently, are often sophisticated in nature, and may not be detected until after a security incident has occurred. Further, Neurogene may experience delays in developing and deploying remedial measures designed to address any such identified vulnerabilities. Applicable data privacy and security obligations may require Neurogene to notify relevant stakeholders of security incidents. Such disclosures are costly, and the disclosure or the failure to comply with such requirements could lead to adverse consequences.

Neurogene relies on third-party service providers and technologies to operate critical business systems to process sensitive information in a variety of contexts. Its ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place. If its third-party service providers experience a security incident or other interruption, Neurogene could experience adverse consequences. While Neurogene may be entitled to damages if its third-party service providers fail to satisfy their privacy or security-related obligations to Neurogene, any award may be insufficient to cover its monetary, reputational and other damages, or Neurogene may be unable to recover such award. In addition, supply-chain attacks have increased in frequency and severity, and Neurogene cannot guarantee that third parties’ infrastructure in its supply chain or its third-party partners’ supply chains have not been compromised.

If Neurogene (or a third party upon whom Neurogene relies) experiences a security incident or is perceived to have experienced a security incident, Neurogene may experience adverse consequences, such as government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including class claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; interruptions in its operations (including availability of data); increased investigation and compliance costs; financial loss; and other similar harms. Security incidents and attendant consequences may cause Neurogene’s stakeholders (including investors and potential customers) to stop supporting its business, deter new customers from its products, deter patients from participating in clinical trials and negatively impact its ability to grow and operate its business.

Neurogene’s contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in its contracts are sufficient to protect Neurogene from liabilities, damages, or claims related to its data privacy and security obligations. Neurogene cannot be sure that its insurance coverage will be adequate or sufficient to protect Neurogene from or to mitigate liabilities arising out of its privacy and security practices or from disruptions in, or failure or security breach of, its systems or third-party systems where information important to its business operations or commercial development is stored, or that such coverage will continue to be available on commercially reasonable terms or at all, or that such coverage will pay future claims.

Neurogene is subject to stringent and changing laws, regulations and standards, and contractual obligations relating to privacy, data protection, and data security. The actual or perceived failure to comply with such obligations could lead to government enforcement actions (which could include civil or criminal penalties), fines and sanctions, private litigation and/or adverse publicity and could negatively affect its operating results and business.

Neurogene, and third parties with whom Neurogene works, are or may become subject to numerous domestic and foreign laws, regulations, and standards relating to privacy, data protection, and data security, the

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scope of which are changing, subject to differing applications and interpretations, and may be inconsistent among countries, or conflict with other rules. Neurogene is or may become subject to the terms of contractual obligations related to privacy, data protection, and data security. Neurogene’s obligations may also change or expand as its business grows. The actual or perceived failure by Neurogene or third parties related to Neurogene to comply with such laws, regulations and obligations could increase Neurogene’s compliance and operational costs, expose it to regulatory scrutiny, actions, fines and penalties, result in reputational harm, lead to a loss of customers, result in litigation and liability, and otherwise cause a material adverse effect on its business, financial condition, and results of operations. See the sections entitled “Neurogene’s Business—Government Regulation—Data Privacy and Security” and “—Other Regulatory Matters” for a more detailed description of the laws that may affect its ability to operate.

If Neurogene fails to comply with environmental, health and safety laws and regulations, Neurogene could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of its business.

Neurogene is subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Its operations may involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. In addition, Neurogene may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. These current or future laws and regulations may impair Neurogene’s research, development or commercialization efforts. Failure to comply with these laws and regulations also may result in substantial fines, penalties or other sanctions.

Neurogene’s ability to utilize its net operating loss carryforwards and certain other tax attributes may be limited.

As of December 31, 2022, Neurogene had net operating loss carryforwards for federal and state income tax purposes of $110.5 million and $36.0 million, respectively. The federal net operating losses will not be subject to expiration and can be carried forward indefinitely; however, they are limited to a deduction to 80% of annual taxable income. The state net operating losses begin to expire in 2038. To the extent that Neurogene’s taxable income exceeds any current year operating losses, Neurogene plans to use its carryforwards to offset income that would otherwise be taxable. Also, for state income tax purposes, the extent to which states will conform to the federal laws is uncertain and there may be periods during which the use of net operating loss carryforwards are suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. In addition, under Section 382 of the Code, changes in Neurogene’s ownership may limit the amount of its net operating loss carryforwards and tax credit carryforwards that could be utilized annually to offset Neurogene’s future taxable income, if any. This limitation would generally apply in the event of a cumulative change in ownership of Neurogene of more than 50% (as measured by value) among a stockholder or one or more groups of stockholders who own at least 5% of Neurogene’s stock within a three-year period. Neurogene has not performed an analysis to determine whether there has been an ownership change pursuant to Section 382. Any such limitation may significantly reduce Neurogene’s ability to utilize its net operating loss carryforwards and tax credit carryforwards before they expire. Any such limitation, whether as the result of a public offering, private placements, sales of Neurogene’s common stock by its existing stockholders or additional sales of its common stock by Neurogene, could have a material adverse effect on Neurogene’s results of operations in future years.

Neurogene may be subject to adverse legislative or regulatory tax changes that could negatively impact its financial condition.

The rules dealing with U.S. federal, state and local income taxation are constantly under review by persons involved in the legislative process and by the Internal Revenue Service and the U.S. Treasury Department. Changes to tax laws (which changes may have retroactive application) could adversely affect its stockholders or Neurogene. Neurogene assesses the impact of various tax reform proposals and modifications to existing tax

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treaties in all jurisdictions where it has operations to determine the potential effect on its business and any assumptions it has made about its future taxable income. Neurogene cannot predict whether any specific proposals will be enacted, the terms of any such proposals or what effect, if any, such proposals would have on its business if they were to be enacted. For example, the United States recently enacted the Inflation Reduction Act of 2022, which implements, among other changes, a 1% excise tax on certain stock buybacks. In addition, beginning in 2022, the Tax Cuts and Jobs Act eliminates the currently available option to deduct research and development expenditures and requires taxpayers to amortize them generally over five years. The U.S. Congress is considering legislation that would restore the current deductibility of research and development expenditures, however, there is no assurance that the provision will be repealed or otherwise modified. Such changes, among others, may adversely affect its effective tax rate, results of operation and general business condition.

Neurogene may acquire businesses or products, or form strategic alliances, in the future, and may not realize the benefits of such acquisitions.

Neurogene may acquire additional businesses or products, form strategic alliances, or create joint ventures with third parties that Neurogene believes will complement or augment its existing business. If Neurogene acquires businesses with promising markets or technologies, Neurogene may not be able to realize the benefit of acquiring such businesses if Neurogene is unable to successfully integrate them with its existing operations and company culture. Neurogene may encounter numerous difficulties in developing, manufacturing and marketing any new product candidates or products resulting from a strategic alliance or acquisition that delay or prevent Neurogene from realizing their expected benefits or enhancing its business. There is no assurance that, following any such acquisition, Neurogene will achieve the synergies expected in order to justify the transaction, which could result in a material adverse effect on its business and prospects.

Neurogene maintains its cash at financial institutions, at times in balances that exceed federally-insured limits. The failure of financial institutions could adversely affect Neurogene’s ability to pay its operational expenses or make other payments.

Neurogene’s cash held in non-interest-bearing and interest-bearing accounts at financial institutions can at times exceed the Federal Deposit Insurance Corporation (“FDIC”) insurance limits. If such banking institutions were to fail, Neurogene could lose all or a portion of those amounts held in excess of such insurance limitations. For example, the FDIC took control of Silicon Valley Bank on March 10, 2023. The Federal Reserve subsequently announced that account holders would be made whole. However, the FDIC may not make all account holders whole in the event of future bank failures. In addition, even if account holders are ultimately made whole with respect to a future bank failure, account holders’ access to their accounts and assets held in their accounts may be substantially delayed. Any material loss that Neurogene may experience in the future or inability for a material time period to access its cash and cash equivalents could have an adverse effect on its ability to pay its operational expenses or make other payments, which could adversely affect its business.

At the end of August 2023, Neurogene identified a material weakness in its internal control over financial reporting and may identify additional material weaknesses in the future that may cause Neurogene to fail to meet its reporting obligations or result in material misstatements of its financial statements. If Neurogene’s internal control over financial reporting or its disclosure controls and procedures are not effective, it may not be able to accurately report its financial results, prevent fraud or file its periodic reports in a timely manner, which may cause investors to lose confidence in its reported financial information and may lead to a decline in its share price.

Neurogene’s internal controls related to the cash disbursements process were not adequately designed to identify unauthorized payment requests, resulting in the identification of a material weakness. Specifically, at the end of August 2023, Neurogene discovered that it was subject to a business email compromise attack by a third party. This deficiency in Neurogene’s controls resulted in the diversion of payments to a fraudulent bank account.

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Neurogene determined that certain internal controls required for safeguarding its cash assets were not properly designed due to insufficient specificity regarding its policies and procedures surrounding supplier banking information changes, not identifying segregation of duties, and insufficient training on exercising professional skepticism.

There can be no assurance that the remediation plans Neurogene has initiated after discovery of the business email compromise attack will be effective. These plans include the following actions:

Management remains committed to the implementation of remediation efforts to address this material weakness. Neurogene has already begun implementation of remediation efforts and remains committed to completing such remediation efforts, and expects to incur additional costs to do so, although there can be no assurance that Neurogene’s efforts will be successful or that it will be able to avoid potential future material weaknesses. If Neurogene is unable to successfully remediate its existing or any future material weakness in its internal control over financial reporting, or if Neurogene identifies any additional material weaknesses, the accuracy and timing of its financial reporting may be adversely affected, Neurogene may be unable to maintain compliance with securities law requirements regarding timely filing of periodic reports in addition to applicable stock exchange listing requirements, investors may lose confidence in its financial reporting, and its stock price may decline as a result. Neurogene also could become subject to investigations by Nasdaq, the SEC or other regulatory authorities.

Risks Related to Intellectual Property

Neurogene’s ability to protect its patents and other proprietary rights is uncertain, exposing Neurogene to the possible loss of competitive advantage.

Neurogene relies and expects to continue to rely upon a combination of patents, trademarks, trade secret protection and confidentiality agreements to protect the intellectual property related to its product candidates and technologies and to prevent third parties from unfairly competing with it. Neurogene’s success depends in large part on its ability to obtain and maintain patent protection for platform technologies, including its EXACT gene regulation platform, product candidates and their uses, as well as the ability to operate without infringing on or violating the proprietary rights of others. As of September 18, 2023, Neurogene licenses 17 patent applications, including, U.S. patent applications, international patent applications under the Patent Cooperation Treaty (“PCT”) or otherwise, and expects to continue to file patent applications in the United States and abroad related to discoveries and technologies that are important to its business. However, Neurogene may not be able to protect its intellectual property rights throughout the world and the legal systems in certain countries may not favor enforcement or protection of patents, trade secrets and other intellectual property. Filing, prosecuting and defending patents on product candidates worldwide would be prohibitively expensive and Neurogene’s intellectual property rights in some foreign jurisdictions may be less extensive than those in the United States. As such, Neurogene does not have patents in all countries or all major markets and may not be able to obtain patents in all jurisdictions even if it applies for them. Competitors may operate in countries where Neurogene does not have patent protection and could then freely use Neurogene’s technologies and discoveries in such countries to the extent such technologies and discoveries are publicly known or disclosed in countries where patent protection has not been requested.

Neurogene’s intellectual property portfolio is at an early stage. As of September 18, 2023, Neurogene does not own or in-license any issued patents. Neurogene’s pending and future patent applications may not result in patents being issued. Any issued patents may not afford sufficient protection of Neurogene’s product candidates or their intended uses against competitors, nor can there be any assurance that the patents issued will not be

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infringed, designed around, invalidated by third parties, or effectively prevent others from commercializing competitive technologies, products or product candidates. Even if these patents are granted, they may be difficult to enforce. Further, any issued patents that may be licensed or owned covering Neurogene product candidates could be narrowed or found invalid or unenforceable if challenged in court or before administrative bodies in the United States or abroad, including the United States Patent and Trademark Office (“USPTO”). Further, if Neurogene encounters delays in any clinical trials or delays in obtaining regulatory approval, the period of time during which Neurogene could market product candidates under patent protection would be reduced. Thus, the patents that Neurogene may own or license may not afford any meaningful competitive advantage.

In addition to seeking patents for some of its technology and product candidates, Neurogene may also rely on trade secrets, including unpatented know-how, technology and other proprietary information, to maintain its competitive position. Any disclosure, either intentional or unintentional, by its employees, the employees of third parties with whom Neurogene shares facilities or third-party consultants and vendors that Neurogene engages to perform researches, clinical trials or manufacturing activities, or misappropriation by third parties (such as through a cybersecurity breach) of its trade secrets or proprietary information could enable competitors to duplicate or surpass Neurogene’s technological achievements, thus eroding its competitive position in the market. In order to protect its proprietary technology and processes, Neurogene relies in part on confidentiality agreements with collaborators, employees, consultants, outside scientific collaborators and sponsored researchers and other advisors. These agreements may not effectively prevent disclosure of confidential information and may not provide an adequate remedy in the event of unauthorized disclosure of confidential information. Neurogene may need to share its proprietary information, including trade secrets, with future business partners, collaborators, contractors and others located in countries at heightened risk of theft of trade secrets, including through direct intrusion by private parties or foreign actors and those affiliated with or controlled by state actors. In addition, while Neurogene undertakes efforts to protect its trade secrets and other confidential information from disclosure, others may independently discover trade secrets and proprietary information, and in such cases, Neurogene may not be able to assert any trade secret rights against such party. Costly and time-consuming litigation could be necessary to enforce and determine the scope of its proprietary rights and failure to obtain or maintain trade secret protection could adversely affect Neurogene’s competitive business position.

Lastly, if Neurogene’s trademarks and trade names are not registered or adequately protected, then Neurogene may not be able to build name recognition in markets of interest and its business may be adversely affected.

Neurogene may not be successful in obtaining or maintaining necessary rights to product candidates through acquisitions and in-licenses.

Because Neurogene’s development programs require and may in the future require the use of proprietary rights held by third parties, the growth of its business may depend in part on Neurogene’s ability to acquire, in-license, or use these third-party proprietary rights. Neurogene may be unable to acquire or in-license any compositions, methods of use, processes or other third-party intellectual property rights from third parties that it identifies as necessary for product candidates. The licensing and acquisition of third-party intellectual property rights is a competitive area, and a number of more established companies may pursue strategies to license or acquire third-party intellectual property rights that Neurogene may consider attractive or necessary. These established companies may have a competitive advantage over Neurogene due to their size, capital resources and greater clinical development and commercialization capabilities. In addition, companies that perceive Neurogene to be a competitor may be unwilling to assign or license rights to Neurogene. Neurogene also may be unable to license or acquire third-party intellectual property rights on terms that would allow it to make an appropriate return on investment or at all. If Neurogene is unable to successfully obtain rights to required third-party intellectual property rights or maintain the existing intellectual property rights it has, Neurogene may have to abandon development of the relevant product candidate, which could have a material adverse effect on its business, financial condition, results of operations, and prospects.

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While Neurogene will normally seek to obtain the right to control prosecution, maintenance and enforcement of the patents relating to a product candidate, there may be times when the filing and prosecution activities for patents and patent applications relating to a product candidate are controlled by future licensors or collaboration partners. For example, Neurogene currently licenses several patent families from the University of Edinburgh covering its EXACT gene regulation platform, as well as the NGN-401 product candidate and its uses. Neurogene also licenses a patent family covering the NGN-101 product candidate and its uses from UNC. If any of such licensors or collaboration partners fail to prosecute, maintain and enforce such patents and patent applications in a manner consistent with the best interests of Neurogene’s business, including by payment of all applicable fees for patents covering a product candidate, Neurogene could lose rights to the intellectual property or exclusivity with respect to those rights, Neurogene’s ability to develop and commercialize such candidates may be adversely affected and it may not be able to prevent competitors from making, using and selling competing products. In addition, even where Neurogene has the right to control patent prosecution of patents and patent applications which may be licensed to and from third parties, Neurogene may still be adversely affected or prejudiced by actions or inactions of licensees, future licensors and their counsel that took place prior to the date upon which Neurogene assumed control over patent prosecution.

Neurogene’s future licensors may rely on third-party consultants or collaborators or on funds from third parties such that future licensors are not the sole and exclusive owners of the patents Neurogene in-licenses. If other third parties have ownership rights to future in-licensed patents, they may be able to license such patents to Neurogene’s competitors, and the competitors could market competing products and technology. This could have a material adverse effect on Neurogene’s competitive position, business, financial conditions, results of operations, and prospects.

It is possible that Neurogene may be unable to obtain licenses at a reasonable cost or on reasonable terms, if at all. Even if Neurogene is able to obtain a license, it may be non-exclusive, thereby giving competitors access to the same technologies licensed to Neurogene. In that event, Neurogene may be required to expend significant time and resources to redesign its technology, product candidates, or the methods for manufacturing the same, or to develop or license replacement technology, all of which may not be feasible on a technical or commercial basis. If Neurogene is unable to do so, it may be unable to develop or commercialize the affected product candidates, which could harm Neurogene’s business, financial condition, results of operations, and prospects significantly. Neurogene cannot provide any assurances that third-party patents do not exist which might be enforced against Neurogene’s current technology or manufacturing methods, its product candidates, or future methods or product candidates, resulting in either an injunction prohibiting manufacture or future sales, or, with

respect to future sales, an obligation on Neurogene’s part to pay royalties and/or other forms of compensation to third parties, which could be significant.

Disputes may arise between Neurogene and its future licensors regarding intellectual property subject to a license agreement, including: the scope of rights granted under the license agreement and other interpretation-related issues; whether and to what extent to which Neurogene’s technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; Neurogene’s right to sublicense patents and other rights to third parties; Neurogene’s right to transfer or assign the license; the inventorship and ownership of inventions and know-how resulting from the joint creations or use of intellectual property by future licensors and Neurogene and/or its partners; and the priority date of an invention of patented technology.

Certain of Neurogene’s current product candidates and research programs are licensed from or based upon licenses from a third party and are field limited to certain indications. If these license agreements are terminated or interpreted to narrow Neurogene’s rights, Neurogene’s ability to advance its current product candidates or develop new product candidates based on these technologies will be materially adversely affected.

Neurogene depends on, and will continue to depend on, its current licenses with UNC, the University of Edinburgh, Virovek, Inc. (“Virovek”) and Sigma-Aldrich Co. LLC (“Sigma”), and on licenses and sublicenses

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from other third parties, as well as potentially on other strategic relationships with third parties, for the research, development, manufacturing and commercialization of Neurogene’s current product candidates. If any of Neurogene’s licenses or relationships or any in-licenses on which its licenses are based are terminated or breached, it may:

Additionally, even if not terminated or breached, Neurogene’s intellectual property licenses or sublicenses may be subject to disagreements over contract interpretation which could narrow the scope of Neurogene’s rights to the relevant intellectual property or technology or increase Neurogene’s financial or other obligations.

If Neurogene experiences any of the foregoing, it could have a materially adverse effect on its business and could force Neurogene to cease operations.

If Neurogene fails to comply with its obligations in any agreements under which it may license intellectual property rights from third parties or otherwise experience disruptions to its business relationships with its licensors, it could lose license rights that are important to its business.

Neurogene is party to license agreements with UNC, the University of Edinburgh, Virovek and Sigma and it may from time to time in the future be party to other license and collaboration agreements with third parties to advance its research or allow commercialization of current or future product candidates. Such agreements may impose numerous obligations, such as development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations on Neurogene and may require Neurogene to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses. See “Neurogene’s Business—License Agreements” for more information regarding Neurogene’s license agreements with UNC, the University of Edinburgh, Virovek and Sigma. Despite Neurogene’s best efforts, its licensors might conclude that it has materially breached its license agreements and might therefore terminate the license agreements, thereby removing or limiting its ability to develop and commercialize products and technologies covered by these license agreements.

If these licenses are terminated for any reason, or if the underlying patents fail to provide the intended exclusivity, Neurogene could lose significant rights and its ability to commercialize its current or future product candidates may be harmed, and competitors or other third parties would have the freedom to seek regulatory approval of, and to market, products identical to Neurogene’s and Neurogene may be required to cease its development and commercialization of certain of its current or future product candidates. Any of the foregoing could have a material adverse effect on Neurogene’s competitive position, business, financial conditions, results of operations, and prospects.

Disputes may also arise between Neurogene and its licensors regarding intellectual property subject to a license agreement, including:

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In addition, the agreements under which Neurogene may license intellectual property or technology from third parties are likely to be complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what Neurogene believes to be the scope of its rights to the relevant intellectual property or technology, or increase what it believes to be its financial or other obligations under the relevant agreement, either of which could have a material adverse effect on its business, financial condition, results of operations and prospects. Moreover, if disputes over intellectual property that Neurogene may license prevent or impair its ability to maintain future licensing arrangements on acceptable terms, it may be unable to successfully develop and commercialize the affected current or future product candidates, which could have a material adverse effect on its business, financial conditions, results of operations and prospects.

Neurogene may be subject to patent infringement claims or may need to file claims to protect its intellectual property, which could result in substantial costs, liability and diversion of resources, and prevent or delay Neurogene from commercializing potential products.

Because the intellectual property landscape in the biotechnology industry is rapidly evolving and interdisciplinary, it is difficult to conclusively assess Neurogene’s freedom to operate and guarantee that it can operate without infringing on or violating third party rights. If certain of Neurogene’s product candidates are ultimately granted regulatory approval, patent rights held by third parties, if found to be valid and enforceable, could be alleged to render one or more of such product candidates infringing. Neurogene cannot be certain that patents owned or licensed by it will not be challenged by others in the course of litigation. If a third party successfully brings a claim against Neurogene, Neurogene may be required to pay substantial damages, be forced to abandon any affected product candidate and/or seek a license from the patent holder. In addition, any uncertainties resulting from the initiation and continuation of any litigation could have a material adverse effect on Neurogene’s business.

Competitors may infringe or otherwise violate Neurogene’s patents, trademarks, copyrights or other intellectual property. To counter infringement or other violations, Neurogene may be required to file claims, which can be expensive and time-consuming. Any such claims could provoke these parties to assert counterclaims against Neurogene, including claims alleging that Neurogene’s intellectual property, methods or products infringes their patents or other intellectual property rights. In addition, in a patent infringement proceeding, a court or administrative body may decide that one or more of the patents Neurogene asserts is invalid or unenforceable, in whole or in part, construe the patent’s claims narrowly or refuse to prevent the other party from using the technology at issue on the grounds that Neurogene’s patents do not cover the technology. Similarly, if Neurogene asserts trademark infringement claims, a court or administrative body may determine that the marks asserted are invalid or unenforceable or that the party against whom Neurogene has asserted trademark infringement has superior rights to the marks in question. In such a case, Neurogene could ultimately be forced to cease use of such marks. In any intellectual property litigation, even if Neurogene is successful, any award of monetary damages or other remedy received may not be commercially valuable.

Further, Neurogene may be required to protect its patents through procedures created to attack the validity of a patent at the USPTO. An adverse determination in any such submission or proceeding could reduce the

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scope or enforceability of, or invalidate, Neurogene’s patent rights, which could adversely affect its competitive position. Because of a lower evidentiary standard in USPTO proceedings compared to the evidentiary standard in U.S. federal courts necessary to invalidate a patent claim, a third party could potentially provide evidence in a USPTO proceeding sufficient for the USPTO to hold a claim invalid even though the same evidence would be insufficient to invalidate the claim if first presented in a district court action.

If Neurogene is required to defend intellectual property actions brought by third parties, or if Neurogene sues to protect its own intellectual property rights or otherwise to protect is proprietary information and to prevent its disclosure, or if Neurogene is involved in other litigation, whether as a plaintiff or defendant, and whether or not successful, Neurogene may incur substantial legal expenses and the attention of its management and key personnel may be diverted from business operations. Further, some of Neurogene’s competitors may be able to sustain the costs of complex intellectual property litigation more effectively than Neurogene can because they have substantially greater resources.

In addition, if Neurogene’s product candidates are found to infringe the intellectual property rights of third parties, these third parties may assert infringement claims against Neurogene’s future licensees and other parties with whom it has business relationships and Neurogene may be required to indemnify those parties for any damages they suffer as a result of these claims, which may require Neurogene to initiate or defend protracted and costly litigation on behalf of licensees and other parties regardless of the merits of such claims. If any of these claims succeed, Neurogene may be forced to pay damages on behalf of those parties or may be required to obtain licenses for the products they use, and may not be able to obtain such licenses on terms acceptable to Neurogene, if at all.

Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation or other legal proceedings relating to Neurogene’s intellectual property rights, there is a risk that some of Neurogene’s confidential information could be compromised by disclosure during this type of litigation or other proceedings.

Neurogene may be subject to claims that it has wrongfully hired an employee from a competitor or that employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.

As is common in the biotechnology industry, in addition to employees, Neurogene engages and may engage in the services of consultants to assist in the development of its product candidates. Many of these consultants, and many of Neurogene’s employees, were or may have been previously employed at, or may have previously provided or may be currently providing consulting services to, other biotechnology or pharmaceutical companies including Neurogene’s competitors or potential competitors. Neurogene could in the future be subject to claims that it or its employees have inadvertently or otherwise used or disclosed alleged trade secrets or other confidential information of former employers or competitors. Although Neurogene tries to ensure that its employees and consultants do not use the intellectual property, proprietary information, know-how or trade secrets of others in their work for Neurogene, Neurogene may become subject to claims that it caused an employee to breach the terms of his or her non-competition or non-solicitation agreement, or that Neurogene or these individuals have, inadvertently or otherwise, used or disclosed the alleged trade secrets or other proprietary information of a former employer or competitor.

Neurogene may litigate to defend itself against these claims, and even if it is successful, litigation could result in substantial costs and could be a distraction to management. If Neurogene’s defenses to these claims fail, in addition to requiring Neurogene to pay monetary damages, a court could prohibit it from using technologies or features that are essential to Neurogene’s product candidates, if such technologies or features are found to incorporate or be derived from the trade secrets or other proprietary information of the former employers. Moreover, any such litigation or the threat thereof may adversely affect Neurogene’s reputation, its ability to form strategic alliances or sublicense its rights to collaborators, engage with scientific advisors or hire employees

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or consultants, each of which would have an adverse effect on Neurogene’s business, its operations and financial condition.

Changes to patent laws in the United States and other jurisdictions could diminish the value of patents in general, thereby impairing Neurogene’s ability to protect its products.

Changes in either the patent laws or interpretation of patent laws in the United States, including patent reform legislation such as the Leahy-Smith America Invents Act (the “Leahy-Smith Act”) could increase the uncertainties and costs surrounding the prosecution of Neurogene’s owned and any future in-licensed patent applications and the maintenance, enforcement or defense of Neurogene’s owned and any future in-licensed issued patents. The Leahy-Smith Act includes a number of significant changes to U.S. patent law. These changes include provisions that affect the way patent applications are prosecuted, redefine prior art, provide more efficient and cost-effective avenues for competitors to challenge the validity of patents, and enable third-party submission of prior art to the USPTO during patent prosecution along with additional procedures to attack the validity of a patent at USPTO-administered post-grant proceedings, including post-grant review, inter partes review, and derivation proceedings. Assuming that other requirements for patentability are met, prior to March 16, 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent. After March 16, 2013, under the Leahy-Smith Act, the United States transitioned to a first-to-file system in which, assuming that the other statutory requirements for patentability are met, the first inventor to file a patent application will be entitled to the patent on an invention regardless of whether a third party was the first to invent the claimed invention. As such, the Leahy-Smith Act and its implementation could increase the uncertainties and costs surrounding the prosecution of Neurogene’s patent applications and the enforcement or defense of Neurogene’s issued patents, all of which could have a material adverse effect on its business, financial condition, its operations and prospects.

In addition, the patent positions of companies in the development and commercialization of biologics and pharmaceuticals are particularly uncertain. U.S. Supreme Court and U.S. Court of Appeals for the Federal Circuit rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations, including in the antibody arts. This combination of events has created uncertainty with respect to the validity and enforceability of patents once obtained. Depending on future actions by the U.S. Congress, the federal courts and the USPTO, the laws and regulations governing patents could change in unpredictable ways that could have a material adverse effect on Neurogene’s patent rights and its ability to protect, defend and enforce Neurogene’s patent rights in the future.

Geopolitical actions in the United States and in foreign countries could increase the uncertainties and costs surrounding the prosecution or maintenance of patent applications and the maintenance, enforcement or defense of issued patents. Accordingly, Neurogene’s competitive position may be impaired, and its business, financial condition, operations and prospects may be adversely affected.

In addition, a European Unified Patent Court (“UPC”) came into force in June 2023. The UPC is a common patent court to hear patent infringement and revocation proceedings effective for member states of the European Union. This could enable third parties to seek revocation of a European patent in a single proceeding at the UPC rather than through multiple proceedings in each of the jurisdictions in which the European patent is validated. Neurogene currently has three pending European applications, and if Neurogene obtains such patents and applications in the future, any such revocation and loss of patent protection could have a material adverse impact on Neurogene’s business and its ability to commercialize or license its technology and products. Moreover, the controlling laws and regulations of the UPC will develop over time, and may adversely affect Neurogene’s ability to enforce or defend the validity of any European patents obtained. Neurogene may decide to opt out from the UPC for any future European patent applications that it may file and any patents it may obtain. If certain formalities and requirements are not met, however, such European patents and patent applications could be challenged for non-compliance and brought under the jurisdiction of the UPC. Neurogene cannot be certain that future European patents and patent applications will avoid falling under the jurisdiction of the UPC, if Neurogene decides to opt out of the UPC.

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Obtaining and maintaining patent protection depends on compliance with various procedural, document submissions, fee payment and other requirements imposed by governmental patent agencies, and Neurogene’s patent protection could be reduced or eliminated for non-compliance with these requirements.

Periodic maintenance fees, renewal fees, annuities fees and various other governmental fees on patents and/or patent applications are due to be paid to the USPTO and foreign patent agencies in several stages over the lifetime of the patent and/or patent application. The USPTO and various foreign governmental patent agencies also require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process. While an inadvertent lapse can in many cases be cured by payment of a late fee or by other means in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. Non-compliance events that could result in abandonment or lapse of a patent or patent application include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. If Neurogene fails to maintain the patents and patent applications covering its product candidates, Neurogene’s competitive position would be adversely affected.

Neurogene may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might adversely affect Neurogene’s ability to develop and market its products.

Neurogene cannot guarantee that any of its patent searches or analyses, including the identification of relevant patents, the scope of patent claims or the expiration of relevant patents, are complete or thorough, nor can Neurogene be certain that it has identified each and every third-party patent and pending application in the United States and abroad that is relevant to or necessary for the commercialization of its product candidates in any jurisdiction. The scope of a patent claim is determined by an interpretation of the law, the written disclosure in a patent and the patent’s prosecution history. Neurogene’s interpretation of the relevance or the scope of a patent or a pending application may be incorrect. For example, Neurogene may incorrectly determine that its products are not covered by a third-party patent or may incorrectly predict whether a third party’s pending application will issue with claims of relevant scope. Neurogene’s determination of the expiration date of any patent in the United States or abroad that it considers relevant may be incorrect. Neurogene’s failure to identify and correctly interpret relevant patents may negatively impact its ability to develop and market its products.

In addition, because some patent applications in the United States may be maintained in secrecy until the patents are issued, patent applications in the United States and many foreign jurisdictions are typically not published until 18 months after filing, and publications in the scientific literature often lag behind actual discoveries, Neurogene cannot be certain that others have not filed patent applications for technology covered by Neurogene’s pending applications or any future issued patents, or that Neurogene was the first to invent the technology. Neurogene’s competitors may have filed, and may in the future file, patent applications covering its products or technology similar to Neurogene’s. Any such patent application may have priority over Neurogene’s patent applications or patents, which could require Neurogene to obtain rights to issued patents covering such technologies.

Neurogene may become subject to claims challenging the inventorship or ownership of its patents and other intellectual property.

Neurogene may be subject to claims that former employees, collaborators or other third parties have an interest in Neurogene’s patents or other intellectual property as an inventor or co-inventor. The failure to name the proper inventors on a patent application can result in the patents issuing thereon being unenforceable. Inventorship disputes may arise from conflicting views regarding the contributions of different individuals named as inventors, the effects of foreign laws where foreign nationals are involved in the development of the subject matter of the patent, conflicting obligations of third parties involved in developing Neurogene’s product

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candidates or as a result of questions regarding co-ownership of potential joint inventions. Litigation may be necessary to resolve these and other claims challenging inventorship and/or ownership. Alternatively, or additionally, Neurogene may enter into agreements to clarify the scope of its rights in such intellectual property. If Neurogene fails in defending any such claims, in addition to paying monetary damages, Neurogene may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material adverse effect on Neurogene’s business. Even if Neurogene is successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.

Neurogene’s current or future licensors may have relied on third-party consultants or collaborators or on funds from third parties, such as the U.S. government or academic institutions, such that Neurogene’s licensors are not the sole and exclusive owners of the patents Neurogene in-licensed. If other third parties have ownership rights or other rights to Neurogene’s in-licensed patents, they may be able to license such patents to Neurogene’s competitors, and its competitors could market competing products and technology. This could have a material adverse effect on Neurogene’s competitive position, business, financial conditions, operations, and prospects.

Patent terms may be inadequate to protect Neurogene’s competitive position on its product candidates for an adequate amount of time.

Patents have a limited lifespan. In the United States, if all maintenance fees are timely paid, the natural expiration of a patent is generally 20 years from its earliest U.S. non-provisional filing date. Various extensions may be available, but the life of a patent, and the protection it affords, is limited. Even if patents covering Neurogene’s product candidates are obtained, once the patent life has expired, Neurogene may be open to competition from competitive products, including generics or biosimilars. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such product candidates might expire before or shortly after such product candidates are commercialized. As a result, Neurogene’s owned and future licensed patent portfolio may not provide it with sufficient rights to exclude others from commercializing products similar or identical to Neurogene’s.

Some intellectual property that Neurogene has in-licensed may have been discovered through government funded programs and thus may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for U.S.-based companies. Compliance with such regulations may limit Neurogene’s exclusive rights, and limit its ability to contract with non-U.S. manufacturers.

Certain of the intellectual property rights Neurogene has licensed are generated through the use of U.S. government funding and are therefore subject to certain federal regulations. As a result, the U.S. government may have certain rights to intellectual property embodied in Neurogene’s current or future product candidates pursuant to the Bayh-Dole Act of 1980 (the “Bayh-Dole Act”), and implementing regulations. These U.S. government rights in certain inventions developed under a government-funded program include a non-exclusive, non-transferable, irrevocable worldwide license to use inventions for any governmental purpose. In addition, the U.S. government has the right to require Neurogene or its licensors to grant exclusive, partially exclusive, or non-exclusive licenses to any of these inventions to a third party if it determines that: (i) adequate steps have not been taken to commercialize the invention; (ii) government action is necessary to meet public health or safety needs; or (iii) government action is necessary to meet requirements for public use under federal regulations (also referred to as “march-in rights”). The U.S. government also has the right to take title to these inventions if Neurogene, or the applicable licensor, fails to disclose the invention to the government and fails to file an application to register the intellectual property within specified time limits. These time limits have recently been changed by regulation, and may change in the future. Intellectual property generated under a government funded program is also subject to certain reporting requirements, compliance with which may require us or the applicable licensor to expend substantial resources. In addition, the U.S. government requires that any products embodying the subject invention or produced through the use of the subject invention be manufactured substantially in the United States. The manufacturing preference requirement can be waived if the owner of the

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intellectual property can show that reasonable but unsuccessful efforts have been made to grant licenses on similar terms to potential licensees that would be likely to manufacture substantially in the United States or that under the circumstances domestic manufacture is not commercially feasible. This preference for U.S. manufacturers may limit Neurogene’s ability to contract with non-U.S. product manufacturers for products covered by such intellectual property. To the extent any of Neurogene’s current or future intellectual property is generated through the use of U.S. government funding, the provisions of the Bayh-Dole Act may similarly apply.

Risks Related to Government Regulation

The regulatory approval processes of the FDA and other comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable. If Neurogene is not able to obtain, or if there are delays in obtaining, required regulatory approvals for its product candidates, Neurogene will not be able to commercialize, or will be delayed in commercializing, such product candidates, and its ability to generate revenue will be materially impaired.

The process of obtaining regulatory approvals, both in the United States and abroad, is unpredictable, expensive and typically takes many years following commencement of clinical trials, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity and novelty of the product candidates involved. Neurogene cannot commercialize product candidates in the United States without first obtaining regulatory approval from the FDA. Similarly, Neurogene cannot commercialize product candidates outside of the United States without obtaining regulatory approval from comparable foreign regulatory authorities. Before obtaining regulatory approvals for the commercial sale of its product candidates, including its most advanced product candidates, NGN-401 and NGN-101, Neurogene must demonstrate through lengthy, complex and expensive preclinical and clinical trials that such product candidates are safe, pure and effective or potent for each targeted indication. Securing regulatory approval also requires the submission of information about the biological product manufacturing process to, and inspection of manufacturing facilities by, the relevant regulatory authority. Further, a product candidate may not be effective or potent, may be only moderately effective or potent or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude its obtaining marketing approval. The FDA and comparable foreign regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that its data are insufficient for approval and require additional preclinical, clinical or other data. A product candidate could be delayed in receiving, or fail to receive, regulatory approval for many reasons, including: the FDA or comparable foreign regulatory authorities may disagree with the design or implementation of its clinical trials; Neurogene may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is safe, pure, and effective or potent for its proposed indication; the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval; serious and unexpected product-related side effects may be experienced by participants in its clinical trials or by individuals using drugs or biological products similar to a product candidate; Neurogene may be unable to demonstrate that a candidate’s clinical and other benefits outweigh its safety risks; the FDA or comparable foreign regulatory authorities may disagree with its interpretation of data from preclinical studies or clinical trials; the data collected from clinical trials of a product candidate may not be acceptable or sufficient to support the submission of a BLA or other submission or to obtain regulatory approval in the United States or elsewhere, and Neurogene may be required to conduct additional clinical trials; the FDA or the applicable foreign regulatory authority may disagree regarding the formulation, labeling and/or the specifications of a product candidate; the FDA or comparable foreign regulatory authorities may fail to approve the manufacturing processes or facilities of third-party manufacturers with which Neurogene contracts for clinical and commercial supplies; and the approval policies or regulations of the FDA or comparable foreign regulatory authorities may significantly change in a manner rendering Neurogene’s clinical data insufficient for approval.

Of the large number of products in development, only a small percentage successfully complete the FDA or foreign regulatory approval processes and are commercialized. The lengthy approval process as well as the

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unpredictability of future clinical trial results may result in Neurogene failing to obtain regulatory approval to market NGN-401 or NGN-101 or other product candidates, which would significantly harm its business, results of operations and prospects.

If Neurogene were to obtain approval, regulatory authorities may approve any such product candidate for fewer or more limited indications than Neurogene requests, including failing to approve the most commercially promising indications, may grant approval contingent on the performance of costly post-marketing clinical trials, or may approve a product candidate with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that product candidate. If Neurogene is not able to obtain, or if there are delays in obtaining, required regulatory approvals for a product candidate, Neurogene will not be able to commercialize, or will be delayed in commercializing, such product candidate and its ability to generate revenue may be materially impaired.

Because gene therapy is novel and the regulatory landscape that governs any product candidates Neurogene may develop is rigorous, complex, uncertain and subject to change, Neurogene cannot predict the time and cost of obtaining regulatory approval, if received at all, for any product candidates Neurogene may develop.

The regulatory requirements that will govern any novel gene therapy product candidates Neurogene develops are not entirely clear and are subject to change. Within the broader genetic medicine field, very few therapeutic products have received marketing authorization from the FDA or the EMA. Even with respect to more established products that fit into the categories of gene therapies or cell therapies, the regulatory landscape is still developing. Regulatory requirements governing gene therapy products and cell therapy products have changed frequently and will likely continue to change in the future. Moreover, there is substantial overlap in those responsible for review and regulation of existing gene therapy products and cell therapy products. For example, in the United States, the FDA has established the Office of Therapeutic Products within its Center for Biologics Evaluation and Research (“CBER”), as part of its reorganization of the Office of Tissues and Advanced Therapies, to consolidate the review of gene therapy and related products. In addition, the Cellular, Tissue and Gene Therapies Advisory Committee advises CBER on its review.

Neurogene’s product candidates will need to meet safety, purity and efficacy or potency standards applicable to any new biologic under the regulatory framework administered by the FDA. In addition to FDA oversight and oversight by IRBs under guidelines promulgated by the National Institutes of Health (“NIH”) gene therapy clinical trials are also subject to review and oversight by an institutional biosafety committee (“IBC”), a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules at that institution. The IBC assesses the safety of the research and identifies any potential risk to public health or the environment. While the NIH guidelines are not mandatory unless the research in question is being conducted at or sponsored by institutions receiving NIH funding of recombinant or synthetic nucleic acid molecule research, many companies and other institutions not otherwise subject to the NIH guidelines voluntarily follow them. Although the FDA decides whether individual gene therapy protocols may proceed, the review process and determinations of other reviewing bodies can impede or delay the initiation of a clinical trial, even if the FDA has reviewed the trial and approved its initiation.

The same applies in the European Union. The EMA’s Committee for Advanced Therapies (“CAT”) is responsible for assessing the quality, safety, and efficacy of advanced-therapy medicinal products. Advanced-therapy medicinal products include gene therapy medicines, somatic-cell therapy medicines and tissue-engineered medicines. The role of the CAT is to prepare a draft opinion on an application for marketing authorization for a gene therapy medicinal candidate that is submitted to the EMA. In the European Union, the development and evaluation of a gene therapy product must be considered in the context of the relevant EU guidelines. The EMA may issue new guidelines concerning the development and marketing authorization for gene therapy products and require that Neurogene comply with these new guidelines. As a result, the procedures and standards applied to gene therapy products and cell therapy products may be applied to any gene therapy product candidate Neurogene may develop, but that remains uncertain at this point.

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Adverse developments in preclinical studies or clinical trials conducted by others in the field of gene therapy and gene regulation products may cause the FDA, the EMA, and other regulatory authorities to revise the requirements for approval of any product candidates Neurogene may develop or limit the use of products utilizing gene regulation technologies, either of which could harm Neurogene’s business. In addition, the clinical trial requirements of the FDA, the EMA, and other regulatory authorities and the criteria these regulators use to determine the safety, purity and efficacy or potency of a product candidate vary substantially according to the type, complexity, novelty, and intended use and market of the potential products. The regulatory approval process for product candidates such as those being developed by Neurogene can be more expensive and take longer than for other, better known, or more extensively studied pharmaceutical or other product candidates. Further, as Neurogene is developing novel potential treatments for diseases in which, in some cases, there is little clinical experience with potential new endpoints and methodologies, heightened risk that the FDA, the EMA or other regulatory authorities may not consider the clinical trial endpoints to provide clinically meaningful results, and the resulting clinical data and results may be more difficult to analyze. In addition, Neurogene may not be able to identify or develop appropriate animal disease models to enable or support planned clinical development. Any natural history studies that Neurogene may conduct or rely upon in its clinical development may not be accepted by the FDA, EMA or other regulatory authorities. Regulatory agencies administering existing or future regulations or legislation may not allow production and marketing of products utilizing gene regulation technology in a timely manner or under technically or commercially feasible conditions. In addition, regulatory action or private litigation could result in expenses, delays, or other impediments to Neurogene’s research programs or the commercialization of resulting products. Further, approvals by one regulatory agency may not be indicative of what other regulatory agencies may require for approval.

The regulatory review committees and advisory groups described above and the new guidelines they promulgate may lengthen the regulatory review process, require Neurogene to perform additional preclinical studies or clinical trials, increase Neurogene’s development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates, or lead to significant post-approval limitations or restrictions. As Neurogene advances its research programs and develops future product candidates, it will be required to consult with these regulatory and advisory groups and to comply with applicable guidelines. If Neurogene fails to do so, it may be required to delay or discontinue development of any product candidates it identifies and develops. These additional processes may result in a review and approval process that is longer than Neurogene otherwise would have expected. Delays as a result of an increased or lengthier regulatory approval process or further restrictions on the development of Neurogene’s product candidates can be costly and could negatively impact Neurogene’s ability to complete clinical trials and commercialize its current and future product candidates in a timely manner, if at all.

Disruptions at the FDA and other regulatory authorities could negatively affect the review of Neurogene’s regulatory submissions, which could negatively impact its business.

The ability of the FDA and other regulatory authorities to review and approve regulatory submissions can be affected by a variety of factors, including understaffing, disruptions caused by government shutdowns and public health crises. Such disruptions could significantly impact the ability of the FDA or other regulatory authorities to timely review and process its regulatory submissions, which could have a material adverse effect on Neurogene’s business.

Neurogene may not be able to meet requirements for the chemistry, manufacturing and control of its product candidates.

In order to receive approval of its products by the FDA and comparable foreign regulatory authorities, Neurogene must show that Neurogene and its contract manufacturing partners are able to characterize, control and manufacture its biological products safely and in accordance with regulatory requirements. This includes manufacturing the drug substance, developing an acceptable formulation, performing tests to adequately characterize the formulated product, documenting a repeatable manufacturing process, and demonstrating that its

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biological products meet stability requirements. Meeting these chemistry, manufacturing and control (“CMC”) requirements is a complex task that requires specialized expertise. If Neurogene is not able to meet the CMC requirements, Neurogene may not be successful in getting its products approved.

Neurogene intends to deliver its product candidates via a drug delivery device that will have its own regulatory, development, supply and other risks.

Neurogene intends to deliver its product candidates via a drug delivery device, such as a catheter or other delivery system. There may be unforeseen technical complications related to the development activities required to bring such a product to market, including primary container compatibility and/or dose volume requirements. Neurogene’s product candidates may not be approved or may be substantially delayed in receiving approval if the devices do not gain and/or maintain their own regulatory approvals or clearances. Where approval of the drug product and device is sought under a single application, the increased complexity of the review process may delay approval. In addition, some drug delivery devices are provided by single-source unaffiliated third-party companies. Neurogene may be dependent on the sustained cooperation and effort of those third-party companies both to supply the devices and, in some cases, to conduct the studies required for approval or other regulatory clearance of the devices. Even if approval is obtained, Neurogene may also be dependent on those third-party companies continuing to maintain such approvals or clearances once they have been received. Failure of third-party companies to supply the devices, to successfully complete studies on the devices in a timely manner, or to obtain or maintain required approvals or clearances of the devices could result in increased development costs, delays in or failure to obtain regulatory approval and delays in product candidates reaching the market or in gaining approval or clearance for expanded labels for new indications.

Neurogene currently and may in the future conduct clinical trials for its product candidates at sites outside the United States, and the FDA may not accept data from trials conducted in such locations.

Neurogene plans to conduct clinical trials outside the United States, including in Australia, the UK, Europe or other foreign jurisdictions. For example, Neurogene currently intends to conduct its Phase 1/2 clinical trial for NGN-401 in the United States and outside the United States. Neurogene’s Phase 1/2 clinical trial for NGN-101 is currently being conducted in the United States and in the UK. In cases where data from clinical trials conducted outside the United States are intended to serve as the sole basis for marketing approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the United States population and United States medical practice; (ii) the trials were performed by clinical investigators of recognized competence and (iii) the data may be considered valid without the need for an on-site inspection by the FDA or, if the FDA considers such an inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means. Additionally, the FDA’s clinical trial requirements, including sufficient size of patient populations and statistical powering, must be met. Many foreign regulatory authorities have similar approval requirements. In addition, such foreign trials would be subject to the applicable local laws of the foreign jurisdictions where the trials are conducted. There can be no assurance that the FDA or any similar foreign regulatory authority will accept data from trials conducted outside of the United States or the applicable jurisdiction. If the FDA or any similar foreign regulatory authority does not accept such data, it would result in the need for additional trials, which would be costly and time-consuming and delay aspects of Neurogene’s business plan, and which may result in Neurogene’s product candidates not receiving approval or clearance for commercialization in the applicable jurisdiction. Even if the FDA accepts such data, it could require Neurogene to modify its planned clinical trials to receive clearance to initiate such trials in the United States or to continue such trials once initiated.

Other risks inherent in conducting international clinical trials include: foreign regulatory requirements, differences in healthcare services, and differences in cultural customs that could restrict or limit its ability to conduct its clinical trials; administrative burdens of conducting clinical trials under multiple sets of foreign regulations; foreign exchange fluctuations; diminished protection of intellectual property in some countries; and political and economic risks relevant to foreign countries.

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Neurogene’s product candidates for which it intends to seek approval as biologics may face competition sooner than anticipated.

The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act (collectively, the “ACA”), includes a subtitle called the Biologics Price Competition and Innovation Act of 2009 (“BPCIA”), which created an abbreviated approval pathway for biological products that are biosimilar to or interchangeable with an FDA-licensed reference biological product. Under the BPCIA, an application for a highly similar or “biosimilar” product may not be submitted to the FDA until four years following the date that the reference product was first approved by the FDA. In addition, the approval of a biosimilar product may not be made effective by the FDA until 12 years from the date on which the reference product was first approved. During this 12-year period of exclusivity, another company may still market a competing version of the reference product if the FDA approves a full BLA for the competing product containing the sponsor’s own preclinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity and potency of its product.

Neurogene’s investigational biological products, if approved, could be considered reference products entitled to 12-year exclusivity. However, there is a risk that this exclusivity could be shortened due to congressional action or otherwise, or that the FDA will not consider a product candidate to be reference products for competing products, potentially creating the opportunity for competition sooner than anticipated. Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. Moreover, the extent to which a biosimilar, once approved, will be substituted for any reference products in a way that is similar to traditional generic substitution for non-biological products is not yet clear, and will depend on a number of marketplace and regulatory factors that are still developing.

Even if Neurogene receives regulatory approval of NGN-401 or NGN-101 or other product candidates, Neurogene will be subject to extensive ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense and Neurogene may be subject to penalties if it fails to comply with regulatory requirements or experiences unanticipated problems with its product candidates.

Any regulatory approvals that Neurogene may receive for NGN-401 or NGN-101 or other product candidates will require the submission of reports to regulatory authorities and surveillance to monitor the safety, purity and efficacy or potency of such product candidates, may contain significant limitations related to use restrictions for specified age groups, warnings, precautions or contraindications, and may include burdensome post-approval study or risk management requirements. For example, the FDA may require a risk evaluation and mitigation strategy in order to approve a product candidate, which could entail requirements for a medication guide, physician training and communication plans or additional elements to ensure safe use, such as restricted distribution methods, patient registries and other risk minimization tools. In addition, if the FDA or comparable foreign regulatory authorities approve a product candidate, the products and the activities associated with their development and commercialization, including their design, testing, manufacture, safety, purity, efficacy or potency, recordkeeping, labeling, storage, approval, advertising, promotion, sale, distribution, import and export will be subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by comparable foreign regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration, as well as on-going compliance with current cGMPs and GCPs for any clinical trials that Neurogene conducts following approval. In addition, manufacturers of drug products and their facilities are subject to continual review and periodic, unannounced inspections by the FDA and other regulatory authorities for compliance with cGMPs.

If Neurogene or a regulatory authority discovers previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facilities where the product is manufactured, a regulatory authority may impose restrictions on that product, the manufacturing facility or Neurogene, including requiring recall or withdrawal of the product from the market or suspension of manufacturing, requiring the addition of labeling statements, such as a “black box” warning or a contraindication,

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requiring creation of a medication guide outlining the risk of such side effects for distribution to patients, withdrawal or suspension of existing approvals or licenses, refusal to approve pending applications or supplements, restrictions on its ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials, restrictions on the manufacturing process, warning or untitled letters, civil and criminal penalties, injunctions, product seizures, detentions or import bans, voluntary or mandatory publicity requirements and imposition of restrictions on operations, including costly new manufacturing requirements. The occurrence of any event or penalty described above may inhibit Neurogene’s ability to commercialize NGN-401 or NGN-101 or other product candidates and generate revenue and could require Neurogene to expend significant time and resources in response and could generate negative publicity.

Neurogene may face difficulties from healthcare legislative reform measures.

Existing regulatory policies may change, and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of NGN-401 or NGN-101 or other product candidates. Neurogene cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. If Neurogene is slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if it is not able to maintain regulatory compliance, Neurogene may lose any marketing approval that it may have obtained and it may not achieve or sustain profitability. See the section entitled “Neurogene’s Business—Government Regulation—Healthcare Reform” for a more detailed description of healthcare reforms measures that may prevent Neurogene from being able to generate revenue, attain profitability, or commercialize product candidates.

Neurogene’s business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payors, patient organizations and customers will be subject to applicable healthcare regulatory laws, which could expose Neurogene to penalties.

Neurogene’s business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payors, patient organizations and customers may expose Neurogene to broadly-applicable fraud and abuse and other healthcare laws and regulations. These laws may constrain the business or financial arrangements and relationships through which Neurogene conducts its operations, including how Neurogene researches, markets, sells and distributes its product candidates, if approved. See the section entitled “Neurogene’s Business—Government Regulation—Other Healthcare Laws and Compliance Requirements” for a more detailed description of the laws that may affect its ability to operate.

Ensuring that its internal operations and future business arrangements with third parties comply with applicable healthcare laws and regulations will involve substantial costs. If Neurogene’s operations are found to be in violation of any of these laws or any other governmental laws and regulations that may apply to it, Neurogene may be subject to significant penalties, including civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, individual imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of its operations. Further, defending against any such actions can be costly and time-consuming and may require significant personnel resources. Therefore, even if Neurogene is successful in defending against any such actions that may be brought against Neurogene, its business may be impaired.

Even if Neurogene is able to commercialize NGN-401 or NGN-101 or other product candidates, due to unfavorable pricing regulations and/or third-party coverage and reimbursement policies, Neurogene may not be able to offer such products at competitive prices which would seriously harm its business.

Neurogene intends to seek approval to market NGN-401 and NGN-101 and other product candidates in both the United States and in selected foreign jurisdictions. If Neurogene obtains approval in one or more foreign jurisdictions for such product candidates, Neurogene will be subject to rules and regulations in those

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jurisdictions. Its ability to successfully commercialize any product candidates that Neurogene may develop will depend in part on the extent to which reimbursement for these products and related treatments will be available from government health administration authorities, private health insurers and other organizations. Government authorities and other third-party payors, such as private health insurers and health maintenance organizations, decide which medications they will pay for and establish reimbursement levels. Government authorities and other third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. These entities may create preferential access policies for a competitor’s product, including a branded or generic/biosimilar product, over its products in an attempt to reduce their costs, which may reduce its commercial opportunity. Additionally, if any of its product candidates are approved and Neurogene is found to have improperly promoted off-label uses of those programs, Neurogene may become subject to significant liability, which would materially adversely affect its business and financial condition. See the sections entitled “Neurogene’s Business—Government Regulation—Coverage and Reimbursement” and “—Regulation in the European Union” for a more detailed description of the government regulations and third-party payor practices that may affect Neurogene’s ability to commercialize product candidates.

Neurogene is subject to U.S. and certain foreign export and import controls, sanctions, embargoes, anti-corruption laws, and anti-money laundering laws and regulations. Neurogene can face criminal liability and other serious consequences for violations, which can harm its business.

Neurogene is subject to export control and import laws and regulations, including the U.S. Export Administration Regulations, U.S. Customs regulations, various economic and trade sanctions regulations administered by the U.S. Treasury Department’s Office of Foreign Assets Controls, the U.S. Foreign Corrupt Practices Act of 1977, as amended, the U.S. domestic bribery statute contained in 18 U.S.C. § 201, the U.S. Travel Act, the USA PATRIOT Act, and other state and national anti-bribery and anti-money laundering laws in the countries in which Neurogene conducts activities. Anti-corruption laws are interpreted broadly and prohibit companies and their employees, agents, contractors, and other collaborators from authorizing, promising, offering, or providing, directly or indirectly, improper payments or anything else of value to or from recipients in the public or private sector. Neurogene may engage third parties to sell products outside the United States, to conduct clinical trials, and/or to obtain necessary permits, licenses, patent registrations, and other regulatory approvals. Neurogene has direct or indirect interactions with officials and employees of government agencies or government-affiliated hospitals, universities, and other organizations. Neurogene can be held liable for the corrupt or other illegal activities of its employees, agents, contractors, and other collaborators, even if Neurogene does not explicitly authorize or have actual knowledge of such activities. Any violations of the laws and regulations described above may result in substantial civil and criminal fines and penalties, imprisonment, the loss of export or import privileges, debarment, tax reassessments, breach of contract and fraud litigation, reputational harm, and other consequences.

Governments outside the United States may impose strict price controls, which may adversely affect Neurogene’s revenue, if any.

In some countries, particularly member states of the European Union, the pricing of prescription drugs is subject to governmental control. In these countries, pricing negotiations with governmental authorities can take considerable time after receipt of marketing approval for a therapeutic. In addition, there can be considerable pressure by governments and other stakeholders on prices and reimbursement levels, including as part of cost containment measures. Political, economic and regulatory developments may further complicate pricing negotiations, and pricing negotiations may continue after reimbursement has been obtained. Reference pricing used by various EU member states and parallel distribution, or arbitrage between low-priced and high-priced member states, can further reduce prices. To obtain coverage and reimbursement or pricing approvals in some countries, Neurogene or current or future collaborators may be required to conduct a clinical trial or other studies that compare the cost-effectiveness of a product to other available therapies in order to obtain or maintain reimbursement or pricing approval. Publication of discounts by third-party payors or authorities may lead to further pressure on the prices or reimbursement levels within the country of publication and other countries. If

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reimbursement of any product approved for marketing is unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels, Neurogene’s business, financial condition, results of operations or prospects could be materially and adversely affected. Brexit could lead to legal uncertainty and potentially divergent national laws and regulations, including those related to the pricing of prescription pharmaceuticals, as the UK determines which EU laws to replicate or replace. If the UK were to significantly alter its regulations affecting the pricing of prescription pharmaceuticals, Neurogene could face significant new costs.

While Neurogene has received Fast Track designation for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease and Neurogene may seek certain designations for its other product candidates, including Breakthrough Therapy, and Priority Review designations in the United States, Neurogene may not receive such designations, and even if it does, such designations may not lead to a faster development or regulatory review or approval process.

Neurogene has received Fast Track designation in the United States for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease, and Neurogene may seek additional designations for one or more of its other product candidates that could expedite review and approval by the FDA. A Breakthrough Therapy product is defined as a product that is intended, alone or in combination with one or more other products, to treat a serious or life threatening disease or condition, and preliminary clinical evidence indicates that the product may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For products that have been designated as Breakthrough Therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens.

The FDA may also designate a product for Fast Track review if it is intended, whether alone or in combination with one or more other products, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for such a disease or condition. For Fast Track products, sponsors may have greater interactions with the FDA and the FDA may initiate review of sections of a Fast Track product’s application before the application is complete. This rolling review may be available if the FDA determines, after preliminary evaluation of clinical data submitted by the sponsor, that a Fast Track product may be effective.

Neurogene may also seek a priority review designation for one or more of its product candidates. If the FDA determines that a product candidate offers a treatment for a serious condition, and if approved, would provide a significant improvement in safety or effectiveness where no adequate therapy exists, the FDA may designate the product candidate for priority review. A priority review designation means that the goal for the FDA to review an application is six months, rather than the standard review period of ten months.

These designations are within the discretion of the FDA. Accordingly, even if Neurogene believes that one of its product candidates meets the criteria for these designations, the FDA may disagree and instead determine not to make such designation. Further, even if Neurogene receives a designation, the receipt of such designation for a product candidate may not result in a faster development or regulatory review or approval process compared to products considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA, including the Fast Track designation Neurogene received for NGN-401. In addition, even if one or more of Neurogene’s product candidates qualifies for these designations, the FDA may later decide that the product candidates no longer meet the conditions for qualification or decide that the time period for FDA review or approval will not be shortened.

The regenerative medicine advanced therapy, or RMAT, designation by the FDA for any of Neurogene’s product candidates may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that Neurogene’s product candidates will receive marketing approval.

We may seek an RMAT designation for Neurogene’s product candidates if the clinical data support such a designation for one or more product candidates. The RMAT designation program is intended to fulfill the Cures

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Act requirement that the FDA facilitate an efficient development program for, and expedite review of, any product that meets the following criteria: (1) it qualifies as a RMAT, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, with limited exceptions; (2) it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and (3) preliminary clinical evidence indicates that the product has the potential to address unmet medical needs for such a disease or condition. Like breakthrough therapy designation, RMAT designation provides potential benefits that include more frequent meetings with FDA to discuss the development plan for the product candidate, and eligibility for rolling review and priority review. Products granted RMAT designation may also be eligible for accelerated approval on the basis of a surrogate or intermediate endpoint reasonably likely to predict long-term clinical benefit, or may be able to rely upon data obtained from a meaningful number of sites, including through expansion to additional sites. RMAT designation does not change the standards for product approval, and there is no assurance that such designation will result in expedited review or approval or that the approved indication will not be narrower than the indication covered by the RMAT designation. Additionally, RMAT designation can be revoked if the criteria for eligibility cease to be met as clinical data emerges.

Neurogene has received orphan drug designation for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease, and Neurogene may seek orphan drug designation for certain future product candidates, but Neurogene may be unable to obtain such designations or to maintain the benefits associated with orphan drug designation, including market exclusivity, which may cause Neurogene’s revenue, if any, to be reduced.

Neurogene has received orphan drug designation from the FDA for NGN-401 for the treatment of Rett syndrome and has also received orphan drug designation from the FDA and European Medicines Agency for NGN-101 for the treatment of CLN5 Batten disease. Although Neurogene may seek orphan product designation for some or all of its other product candidates, Neurogene may never receive such designations. Under the Orphan Drug Act, the FDA may designate a drug or biological product as an orphan drug if it is intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. Orphan drug designation must be requested before submitting a BLA. In the European Union, the EMA’s Committee for Orphan Medicinal Products grants orphan drug designation to promote the development of products that are intended for the diagnosis, prevention, or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in the European Union. Additionally, designation is granted for products intended for the diagnosis, prevention, or treatment of a life-threatening, seriously debilitating or serious and chronic condition when, without incentives, it is unlikely that sales of the drug in the European Union would be sufficient to justify the necessary investment in developing the drug or biological product or where there is no satisfactory method of diagnosis, prevention, or treatment, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

In the United States, orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages, and application fee waivers. After the FDA grants orphan drug designation, the generic identity of the drug and its potential orphan use are disclosed publicly by the FDA.

In addition, if a product receives the first FDA approval for the indication for which it has orphan designation, the product is entitled to orphan drug exclusivity, which means the FDA may not approve any other application to market the same drug for the same indication for a period of seven years, except in limited circumstances, such as a showing of clinical superiority over the product with orphan exclusivity or where the manufacturer is unable to assure sufficient product quantity for the orphan patient population. Exclusive marketing rights in the United States may also be unavailable if Neurogene or its collaborators seek approval for an indication broader than the orphan designated indication and may be lost if the FDA later determines that the

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request for designation was materially defective. In the European Union, orphan drug designation entitles a party to financial incentives such as reduction of fees or fee waivers and ten years of market exclusivity following drug or biological product approval. This period may be reduced to six years if the orphan drug designation criteria are no longer met, including where it is shown that the product is sufficiently profitable not to justify maintenance of market exclusivity.

Even with an orphan drug designation for its current and potential future product candidates, Neurogene may not be the first to obtain marketing approval for any particular orphan indication due to the uncertainties associated with developing pharmaceutical products. Further, even if Neurogene obtains orphan drug exclusivity for an existing or future product candidate, that exclusivity may not effectively protect the product from competition because different drugs with different active moieties still can be approved for the same condition even with an orphan drug designation. Even after an orphan drug is approved, the FDA can subsequently approve the same drug with the same active moiety for the same condition if the FDA concludes that the later drug is clinically superior in that it is safer, more effective, or makes a major contribution to patient care. Orphan drug designation neither shortens the development time or regulatory review time of a drug or biologic nor gives the drug or biologic any advantage in the regulatory review or approval process.

Neurogene has received Rare Pediatric Disease designation by the FDA for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease. However, Rare Pediatric Disease designation for any of Neurogene’s product candidates does not guarantee that the BLA for the product will qualify for a priority review voucher upon approval, and it does not lead to a faster development or regulatory review process, or increase the likelihood that Neurogene’s product candidates will receive marketing approval.

Under the Rare Pediatric Disease Priority Review Voucher program, upon the approval of a qualifying BLA for the treatment of a rare pediatric disease, the sponsor of such an application would be eligible for a rare pediatric disease priority review voucher that can be used to obtain priority review for a subsequent BLA or NDA. If a product candidate is designated before September 30, 2024, it is eligible to receive a voucher if it is approved before September 30, 2026. While Neurogene has obtained Rare Pediatric Disease designation for NGN-401 for the treatment of Rett syndrome and for NGN-101 for the treatment of CLN5 Batten disease, it is unlikely that these product candidates will be approved by September 30, 2026. If approval is not obtained by then, Neurogene would not be in a position to obtain a priority review voucher, unless Congress further reauthorizes the program beyond the current sunset date in September 2024. Additionally, designation of a biological product for a rare pediatric disease does not guarantee that a BLA will meet the eligibility criteria for a rare pediatric disease priority review voucher at the time the application is approved. Finally, a Rare Pediatric Disease Designation does not lead to faster development or regulatory review of the product or increase the likelihood that it will receive marketing approval.

General Risk Factors

Neurogene’s estimates of market opportunity and forecasts of market growth may prove to be inaccurate, and even if the markets in which Neurogene competes achieve the forecasted growth, its business may not grow at similar rates, or at all.

Neurogene’s market opportunity estimates and growth forecasts are subject to significant uncertainty and are based on assumptions and estimates which may not prove to be accurate. Its estimates and forecasts relating to size and expected growth of its target market may prove to be inaccurate. Even if the markets in which Neurogene competes meet its size estimates and growth forecasts, its business may not grow at similar rates, or at all. Neurogene’s growth is subject to many factors, including its success in implementing its business strategy, which is subject to many risks and uncertainties.

Neurogene’s revenue will be dependent, in part, upon the size of the markets in the territories for which Neurogene gains regulatory approval, the accepted price for the product, the ability to obtain coverage and

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reimbursement and whether Neurogene owns the commercial rights for that territory. If the number of its addressable patients is not as significant as Neurogene estimates, the indication approved by regulatory authorities is narrower than Neurogene expects or the treatment population is narrowed by competition, physician choice or treatment guidelines, Neurogene may not generate significant revenue from sales of such products, even if approved.

Neurogene may become exposed to costly and damaging liability claims, either when testing a product candidate in the clinical or at the commercial stage, and its product liability insurance may not cover all damages from such claims.

Neurogene is exposed to potential product liability and professional indemnity risks that are inherent in the research, development, manufacturing, marketing, and use of pharmaceutical products. While Neurogene currently has no products that have been approved for commercial sale, the current and future use of a product candidate in clinical trials, and the sale of any approved products in the future, may expose Neurogene to liability claims. These claims may be made by patients that use the product, healthcare providers, pharmaceutical companies, or others selling such product. Any claims against Neurogene, regardless of their merit, could be difficult and costly to defend and could materially and adversely affect the market for its products or any prospects for commercialization of its products. Although Neurogene believes it currently maintains adequate product liability insurance for NGN-401 and NGN-101 and other product candidates, it is possible that its liabilities could exceed its insurance coverage or that in the future Neurogene may not be able to maintain insurance coverage at a reasonable cost or obtain insurance coverage that will be adequate to satisfy any liability that may arise. If a successful product liability claim or series of claims is brought against Neurogene for uninsured liabilities or in excess of insured liabilities, its assets may not be sufficient to cover such claims and its business operations could be impaired.

Litigation costs and the outcome of litigation could have a material adverse effect on Neurogene’s business.

From time to time Neurogene may be subject to litigation claims through the ordinary course of its business operations regarding, but not limited to, employment matters, security of patient and employee personal information, contractual relations with collaborators and intellectual property rights. Litigation to defend ourselves against claims by third parties, or to enforce any rights that Neurogene may have against third parties, may continue to be necessary, which could result in substantial costs and diversion of its resources, causing a material adverse effect on its business, financial condition, results of operations or cash flows.

Neurogene’s business could be adversely affected by economic downturns, inflation, increases in interest rates, natural disasters, public health crises such as the COVID-19 pandemic, political crises, geopolitical events, such as conflict between Russia and Ukraine, or other macroeconomic conditions, which could have a material and adverse effect on its results of operations and financial condition.

The global economy, including credit and financial markets, has experienced extreme volatility and disruptions, including, among other things, diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, supply chain shortages, increases in inflation rates, higher interest rates, and uncertainty about economic stability. For example, the COVID-19 pandemic resulted in widespread unemployment, economic slowdown and extreme volatility in the capital markets. The Federal Reserve has raised interest rates multiple times in response to concerns about inflation and it may raise them again. Higher interest rates, coupled with reduced government spending and volatility in financial markets, may increase economic uncertainty and affect consumer spending. Similarly, the ongoing military conflict between Russia and Ukraine and rising tensions with China have created extreme volatility in the global capital markets and may have further global economic consequences, including disruptions of the global supply chain. Any such volatility and disruptions may adversely affect Neurogene’s business or the third parties on whom Neurogene relies. If the equity and credit markets deteriorate, including as a result of political unrest or war, it may make any necessary debt or equity financing more costly, more dilutive, or more difficult to obtain in a timely manner or on favorable

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terms, if at all. Increased inflation rates can adversely affect Neurogene by increasing its costs, including labor and employee benefit costs.

Neurogene may in the future experience disruptions as a result of such macroeconomic conditions, including delays or difficulties in initiating or expanding clinical trials and manufacturing sufficient quantities of materials. Any one or a combination of these events could have a material and adverse effect on Neurogene’s results of operations and financial condition.

Risks Related to the Combined Company

If any of the events described in “Risks Related to Neoleukin” or “Risks Related to Neurogene” occur, those events could cause potential benefits of the merger not to be realized.

Following completion of the merger, the combined company will be susceptible to many of the risks described in the sections herein entitled “Risks Related to Neoleukin” and “Risks Related to Neurogene.” To the extent any of the events in the risks described in those sections occur, the potential benefits of the merger may not be realized and the results of operations and financial condition of the combined company could be adversely affected in a material way. This could cause the market price of the combined company’s common stock to decline.

The market price of the combined company’s common stock is expected to be volatile, and the market price of the common stock may drop following the merger.

The market price of the combined company’s common stock following the merger could be subject to significant fluctuations. Some of the factors that may cause the market price of the combined company’s common stock to fluctuate include:

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Moreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual companies. These broad market fluctuations may also adversely affect the trading price of the combined company’s common stock. In addition, a recession, depression or other sustained adverse market event could materially and adversely affect the combined company’s business and the value of its common stock. In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against such companies. Furthermore, market volatility may lead to increased shareholder activism if the combined company experiences a market valuation that activists believe is not reflective of its intrinsic value. Activist campaigns that contest or conflict with the combined company’s strategic direction or seek changes in the composition of its board of directors could have an adverse effect on its operating results, financial condition and cash flows.

The combined company may incur losses for the foreseeable future and might never achieve profitability.

The combined company may never become profitable, even if the combined company is able to complete clinical development for one or more product candidates and eventually commercialize such product candidates. The combined company will need to successfully complete significant research, development, testing and regulatory compliance activities that, together with projected general and administrative expenses, is expected to result in substantial increased operating losses for at least the next several years. Even if the combined company does achieve profitability, it may not be able to sustain or increase profitability on a quarterly or annual basis.

Following the merger, the combined company may be unable to integrate successfully the businesses of Neoleukin and Neurogene and realize the anticipated benefits of the merger.

The merger involves the combination of two companies which currently operate as independent companies. Following the merger, the combined company will be required to devote significant management attention and resources to integrating its business practices and operations. The combined company may fail to realize some or all of the anticipated benefits of the merger if the integration process takes longer than expected or is more costly than expected. Potential difficulties the combined company may encounter in the integration process include the following:

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In addition, Neoleukin and Neurogene have operated and, until the completion of the merger, will continue to operate, independently. It is possible that the integration process also could result in the diversion of each company’s management’s attention, the disruption or interruption of, or the loss of momentum in, each company’s ongoing businesses or inconsistencies in standards, controls, procedures and policies, any of which could adversely affect the combined company’s ability to maintain its business relationships or the ability to achieve the anticipated benefits of the merger, or could otherwise adversely affect the business and financial results of the combined company.

If the combined company fails to attract and retain management and other key personnel, it may be unable to continue to successfully develop or commercialize its product candidates or otherwise implement its business plan.

The combined company’s ability to compete in the highly competitive pharmaceuticals industry depends on its ability to attract and retain highly qualified managerial, scientific, medical, legal, sales and marketing and other personnel. The combined company will be highly dependent on its management and scientific personnel. The loss of the services of any of these individuals could impede, delay, or prevent the successful development of the combined company’s product pipeline, completion of its planned clinical trials, commercialization of its product candidates or in-licensing or acquisition of new assets and could impact negatively its ability to implement successfully its business plan. If the combined company loses the services of any of these individuals, it might not be able to find suitable replacements on a timely basis or at all, and its business could be harmed as a result. The combined company might not be able to attract or retain qualified management and other key personnel in the future due to the intense competition for qualified personnel among biotechnology, pharmaceutical and other businesses.

The combined company will need to raise additional financing in the future to fund its operations, which may not be available to it on favorable terms or at all.

The combined company will require substantial additional funds to conduct the costly and time-consuming clinical efficacy trials necessary to pursue regulatory approval of each potential product candidate and to continue the development of NGN-401 and NGN-101, Neurogene’s other product candidates and future product candidates. The combined company’s future capital requirements will depend upon a number of factors, including: the number and timing of future product candidates in the pipeline; progress with and results from preclinical testing and clinical trials; the ability to manufacture sufficient drug supplies to complete preclinical and clinical trials; the costs involved in preparing, filing, acquiring, prosecuting, maintaining and enforcing patent and other intellectual property claims; and the time and costs involved in obtaining regulatory approvals and favorable reimbursement or formulary acceptance. Raising additional capital may be costly or difficult to obtain and could, for example, through the sale of common stock or securities convertible or exchangeable into common stock, significantly dilute stockholders’ ownership interests or inhibit the combined company’s ability to achieve its business objectives. If the combined company raises additional funds through public or private equity offerings, the terms of these securities may include liquidation or other preferences that adversely affect the rights of its common stockholders. In addition, any debt financing may subject the combined company to fixed payment obligations and covenants limiting or restricting its ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If the combined company raises additional capital through marketing and distribution arrangements or other collaborations, strategic alliances or licensing arrangements with third parties, the combined company may have to relinquish certain valuable intellectual property or other rights to its product candidates, technologies, future revenue streams or research programs or grant licenses on terms that may not be favorable to it. Even if the combined company were to obtain sufficient funding, there can be no assurance that it will be available on terms acceptable to the combined company or its stockholders.

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If Neoleukin’s legacy lease obligations are not subleased, assigned, terminated or otherwise addressed or the legacy assets subject to the CVR Agreement are not sold, respectively, in a timely manner, the combined company may have to incur time and resources to take such actions.

The combined company will incur additional costs and increased demands upon management as a result of complying with the laws and regulations affecting public companies.

The combined company will incur significant legal, accounting and other expenses as a public company that Neurogene did not incur as a private company, including costs associated with public company reporting obligations under the Securities Exchange Act of 1934, as amended (the “Exchange Act”). The combined company’s management team will consist of the executive officers of Neurogene prior to the merger. These executive officers and other personnel will need to devote substantial time to complying with public company reporting requirements and compliance with applicable laws and regulations to ensure that the combined company complies with all of these requirements. Any changes the combined company makes to comply with these obligations may not be sufficient to allow it to satisfy its obligations as a public company on a timely basis, or at all. These reporting requirements, rules and regulations, coupled with the increase in potential litigation exposure associated with being a public company, could also make it more difficult for the combined company to attract and retain qualified persons to serve on the board of directors or on board committees or to serve as executive officers, or to obtain certain types of insurance, including directors’ and officers’ insurance, on acceptable terms.

Upon completion of the merger, failure by the combined company to comply with the initial listing standards of Nasdaq will prevent its stock from being listed on Nasdaq.

Upon completion of the merger, Neoleukin, under the new name “Neurogene Inc.,” will be required to meet the initial listing requirements to maintain the listing and continued trading of its shares on Nasdaq. These initial listing requirements are more difficult to achieve than the continued listing requirements that currently apply to Neoleukin. Pursuant to the Merger Agreement, Neoleukin agreed to use its commercially reasonable efforts to cause the shares of Neoleukin common stock being issued in the merger to be approved for listing on Nasdaq at or prior to the effective time of the merger. Based on information currently available to Neoleukin, even after giving effect to the 1-for-5 reverse stock split that was effected on September 25, 2023, Neoleukin anticipates that its stock will be unable to meet the $4.00 minimum bid price initial listing requirement at the closing of the

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merger unless it effects a further reverse stock split. The board of directors of Neoleukin, in consultation with the board of directors of Neurogene (assuming the approval by Neoleukin stockholders of Proposal No. 1 (the “Nasdaq Stock Issuance Proposal”)) intends to effect a reverse stock split of the shares of Neoleukin common stock at a ratio of between 1:    to 1:    . However, often times a reverse stock split will not result in a trading price for the affected common stock that is proportional to the ratio of the split. Following the merger, if the combined company is unable to satisfy Nasdaq listing requirements, Nasdaq may notify the combined company that its shares of common stock will not be listed on Nasdaq.

Upon a potential delisting from Nasdaq, if the common stock of the combined company is not then eligible for quotation on another market or exchange, trading of the shares could be conducted in the over-the-counter market or on an electronic bulletin board established for unlisted securities such as the Pink Sheets or the OTC Bulletin Board. In such event, it is likely that there would be significantly less liquidity in the trading of the common stock of the combined company; decreases in institutional and other investor demand for the shares, coverage by securities analysts, market making activity and information available concerning trading prices and volume; and fewer broker dealers willing to execute trades in the common stock of the combined company. Also, it may be difficult for the combined company to raise additional capital if the combined company’s common stock is not listed on a major exchange. The occurrence of any of these events could result in a further decline in the market price of the common stock of the combined company and could have a material adverse effect on the combined company.

Once the combined company is no longer a smaller reporting company or otherwise no longer qualifies for applicable exemptions, the combined company will be subject to additional laws and regulations affecting public companies that will increase the combined company’s costs and the demands on management and could harm the combined company’s operating results and cash flows.

The combined company will be subject to the reporting requirements of the Exchange Act, which requires, among other things, that the combined company file with the SEC annual, quarterly and current reports with respect to the combined company’s business and financial condition as well as other disclosure and corporate governance requirements. Neoleukin and Neurogene expects the combined company to still qualify as a “smaller reporting company,” as such term is defined in Rule 12b-2 under the Exchange Act, in at least the near term, which will allow the combined company to take advantage of many of the same exemptions from disclosure requirements, including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act and reduced disclosure obligations regarding executive compensation in this proxy statement/prospectus and in the combined company’s periodic reports and proxy statements. Once the combined company is no longer a smaller reporting company or otherwise no longer qualifies for this exemption, the combined company will be required to comply with these additional legal and regulatory requirements applicable to public companies and will incur significant legal, accounting and other expenses to do so. If the combined company is not able to comply with the requirements in a timely manner or at all, the combined company’s financial condition or the market price of the combined company’s common stock may be harmed. For example, if the combined company or its independent auditor identifies deficiencies in the combined company’s internal control over financial reporting that are deemed to be material weaknesses, the combined company could face additional costs to remedy those deficiencies, the market price of the combined company’s stock could decline or the combined company could be subject to sanctions or investigations by the SEC or other regulatory authorities, any of which would require additional financial and management resources.

If the combined company fails to maintain proper and effective internal controls, its ability to produce accurate financial statements on a timely basis could be impaired.

Provided any securities of the combined company continue to be registered with the SEC and the combined company continues to be listed on Nasdaq, the combined company will be subject to the reporting requirements of the Exchange Act, the Sarbanes-Oxley Act and the rules and regulations of Nasdaq. The Sarbanes-Oxley Act requires, among other things, that the combined company maintain effective disclosure controls and procedures

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and internal control over financial reporting. The combined company must perform system and process evaluation and testing of its internal control over financial reporting to allow management to report on the effectiveness of its internal controls over financial reporting in its Annual Report on Form 10-K filing for that year, as required by Section 404 of the Sarbanes-Oxley Act. As a private company, Neurogene has never been required to test its internal controls within a specified period. This will require that the combined company incur substantial professional fees and internal costs to expand its accounting and finance functions and that it expends significant management efforts. The combined company may experience difficulty in meeting these reporting requirements in a timely manner. The combined company may discover weaknesses in its system of internal financial and accounting controls and procedures that could result in a material misstatement of its financial statements. The combined company’s internal control over financial reporting will not prevent or detect all errors and all fraud. A control system, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the control system’s objectives will be met. Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud will be detected.

For example, Neurogene’s internal controls related to the cash disbursements process were not adequately designed to identify unauthorized payment requests, resulting in the identification of a material weakness. Specifically, at the end of August 2023, Neurogene discovered that it was subject to a business email compromise attack by a third party. This deficiency in Neurogene’s controls resulted in the diversion of payments to a fraudulent bank account. For more information, see “—At the end of August 2023, Neurogene identified a material weakness in its internal control over financial reporting and may identify additional material weaknesses in the future that may cause Neurogene to fail to meet its reporting obligations or result in material misstatements of its financial statements. If Neurogene’s internal control over financial reporting or its disclosure controls and procedures are not effective, it may not be able to accurately report its financial results, prevent fraud or file its periodic reports in a timely manner, which may cause investors to lose confidence in its reported financial information and may lead to a decline in its share price.” If the combined company is unable to successfully remediate its future material weakness in its internal control over financial reporting, or if the combined company identifies any material weaknesses, the accuracy and timing of its financial reporting may be adversely affected, the combined company may be unable to maintain compliance with securities law requirements regarding timely filing of periodic reports in addition to applicable stock exchange listing requirements, investors may lose confidence in its financial reporting, and its stock price may decline as a result. The combined company also could become subject to investigations by Nasdaq, the SEC or other regulatory authorities.

If the combined company is not able to comply with the requirements of Section 404 of the Sarbanes-Oxley Act, or if it is unable to maintain proper and effective internal controls, the combined company may not be able to produce timely and accurate financial statements. If that were to happen, the market price of its common stock could decline and it could be subject to sanctions or investigations by Nasdaq, the SEC or other regulatory authorities.

The unaudited pro forma condensed combined financial information for Neoleukin and Neurogene included in this proxy statement/prospectus are preliminary, and the combined company’s actual financial position and operations after the merger may differ materially from the unaudited pro forma financial information included in this proxy statement/prospectus.

The unaudited pro forma financial information for Neoleukin and Neurogene included in this proxy statement/prospectus are presented for illustrative purposes only and are not necessarily indicative of the combined company’s actual financial condition or results of operations of future periods, or the financial condition or results of operations that would have been realized had the entities been combined during the period presented. The unaudited pro forma financial information has been derived from the historical financial statements of Neoleukin and Neurogene and adjustments and assumptions have been made regarding the combined company after giving effect to the merger. The information upon which these adjustments and assumptions have been made is preliminary, and these kinds of adjustments and assumptions are difficult to make with accuracy. Moreover, the unaudited pro forma financial information does not reflect all costs that are expected to be incurred by the combined company in connection with

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The combined company’s certificate of incorporation and bylaws, as well as provisions under Delaware law, could make an acquisition of the combined company more difficult and may prevent attempts by its stockholders to replace or remove its management.

If the merger is completed, Neoleukin’s bylaws and Neoleukin’s charter, as amended by the amendments thereto attached to this proxy statement/prospectus as Annex B, assuming Proposal Nos. 2, 3 and 4 are approved by Neoleukin stockholders at the Neoleukin special meeting, will become the combined company’s bylaws and certificate of incorporation. Provisions that will be included in the combined company’s certificate of incorporation and bylaws may discourage, delay or prevent a merger, acquisition or other change in control of the combined company that stockholders may consider favorable, including transactions in which its common stockholders might otherwise receive a premium price for their shares. These provisions could also limit the price that investors might be willing to pay in the future for shares of the combined company’s common stock, thereby depressing the market price of its common stock. In addition, because the combined company’s board of directors will be responsible for appointing the members of the combined company’s management team, these provisions may frustrate or prevent any attempts by the combined company’s stockholders to replace or remove its current management by making it more difficult for stockholders to replace members of the combined company’s board of directors. Among other things, these provisions:

Moreover, because the combined company will be incorporated in Delaware, it is governed by the provisions of Section 203 of the DGCL, which prohibits stockholders owning in excess of 15% of the outstanding combined company voting stock from merging or combining with the combined company. Although Neoleukin and Neurogene believe these provisions collectively will provide for an opportunity to receive higher bids by requiring potential acquirors to negotiate with the combined company’s board of directors, they would apply even if the offer may be considered beneficial by some stockholders. In addition, these provisions may frustrate or prevent any attempts by the combined company’s stockholders to replace or remove then current management by making it more difficult for stockholders to replace members of the board of directors, which is responsible for appointing the members of management.

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The governing documents of the combined company will provide that, unless the combined company consents in writing to the selection of an alternative forum, certain designated courts will be the sole and exclusive forum for certain legal actions between the combined company and its stockholders, which could limit its stockholders’ ability to obtain a favorable judicial forum for disputes with the combined company or its directors, officers, employees or agents.

The governing documents of the combined company will provide that, unless it consents in writing to an alternative forum, the Court of Chancery of the State of Delaware is the sole and exclusive forum for state law claims for (i) any derivative action or proceeding brought on its behalf, (ii) any action asserting a claim of or based on a breach of a fiduciary duty owed by any of its current or former directors, officers, or other employees to the combined company or its stockholders, (iii) any action asserting a claim arising pursuant to any provision of the DGCL, the certificate of incorporation or the bylaws, (iv) any action to interpret, apply, enforce or determine the validity of the certificate of incorporation or bylaws, or (v) any action asserting a claim that is governed by the internal affairs doctrine, in each case subject to the Court of Chancery having personal jurisdiction over the indispensable parties named as defendants therein, which for purposes of this risk factor refers to herein as the “Delaware Forum Provision.” The governing documents of the combined company will further provide that, unless it consents in writing to an alternative forum, the federal district courts of the United States will be the exclusive forum for resolving any complaint asserting a cause of action arising under the Securities Act, which for purposes of this risk factor refers to herein as the “Federal Forum Provision.” Neither the Delaware Forum Provision nor the Federal Forum Provision will apply to any causes of action arising the Exchange Act. In addition, any person or entity purchasing or otherwise acquiring any interest in shares of the combined company’s capital stock will be deemed to have notice of and consented to the foregoing Delaware Forum Provision and Federal Forum Provision; provided, however, that stockholders cannot and will not be deemed to have waived its compliance with the U.S. federal securities laws and the rules and regulations thereunder.

The Delaware Forum Provision and the Federal Forum Provision may impose additional litigation costs on stockholders of the combined company in pursuing any such claims, particularly if the stockholders do not reside in or near the State of Delaware. Additionally, these forum selection clauses may limit its stockholders’ ability to bring a claim in a judicial forum that they find favorable for disputes with the combined company or its directors, officers or employees, which may discourage such lawsuits against the combined company and its directors, officers and employees even though an action, if successful, might benefit its stockholders.

Neoleukin and Neurogene do not anticipate that the combined company will pay any cash dividends in the foreseeable future.

The current expectation is that the combined company will retain its future earnings, if any, to fund the growth of the combined company’s business as opposed to paying dividends. As a result, capital appreciation, if any, of the common stock of the combined company will be your sole source of gain, if any, for the foreseeable future.

An active trading market for the combined company’s common stock may not develop and its stockholders may not be able to resell their shares of common stock for a profit, if at all.

Prior to the merger, there had been no public market for shares of Neurogene capital stock. An active trading market for the combined company’s shares of common stock may never develop or be sustained. If an active market for the combined company’s common stock does not develop or is not sustained, it may be difficult for its stockholders to sell their shares at an attractive price or at all.

Future sales of shares by existing stockholders could cause the combined company’s stock price to decline.

If existing securityholders of Neoleukin and Neurogene sell, or indicate an intention to sell, substantial amounts of the combined company’s common stock in the public market, the trading price of the common stock of the combined company could decline. Based on shares outstanding as of    , 2023, after giving effect to

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the estimated exchange ratio and broad-based weighted average anti-dilution adjustments available to holders of Neurogene Series A-1 Preferred Stock, Series A-2 Preferred Stock and Series B Preferred Stock pursuant to the Neurogene Charter, the shares of Neurogene common stock and the pre-funded warrants to be issued in the Neurogene pre-closing financing and shares expected to be issued upon completion of the merger, the combined company is expected to have outstanding a total of approximately    shares of common stock immediately following the completion of the merger. Of the shares of common stock, approximately     shares will be available for sale in the public market beginning 180 days after the closing of the merger as a result of the expiration of lock-up agreements between Neoleukin and Neurogene on the one hand and certain securityholders of Neoleukin and Neurogene on the other hand. All other outstanding shares of common stock, other than shares held by affiliates of the combined company, will be freely tradable, without restriction, in the public market immediately following the effective time. In addition, shares of common stock that are subject to outstanding options or warrants of Neurogene will become eligible for sale in the public market to the extent permitted by the provisions of various vesting agreements and Rules 144 and 701 under the Securities Act. If these shares are sold, the trading price of the combined company’s common stock could decline.

After completion of the merger, the combined company’s executive officers, directors and principal stockholders will have the ability to control or significantly influence all matters submitted to the combined company’s stockholders for approval.

Upon the completion of the merger, and giving effect to the issuance of the shares of Neurogene common stock and the pre-funded warrants prior to the closing of the merger pursuant to the Neurogene pre-closing financing, it is anticipated that the combined company’s executive officers, directors and principal stockholders will, in the aggregate, beneficially own approximately % of the combined company’s outstanding shares of common stock, subject to certain assumptions, including, but not limited to, Neoleukin’s net cash as of closing being not less than the Target Parent Net Cash amount and receipt by Neurogene of aggregate proceeds of $95.0 million from the Neurogene pre-closing financing. As a result, if these stockholders were to choose to act together, they would be able to control or significantly influence all matters submitted to the combined company’s stockholders for approval, as well as the combined company’s management and affairs. For example, these stockholders, if they choose to act together, would control or significantly influence the election of directors and approval of any merger, consolidation or sale of all or substantially all of the combined company’s assets. This concentration of voting power could delay or prevent an acquisition of the combined company on terms that other stockholders may desire.

The combined company may be exposed to increased litigation, including stockholder litigation, which could have an adverse effect on the combined company’s business and operations.

The combined company may be exposed to increased litigation from stockholders, suppliers and other third parties due to the combination of Neoleukin’s business and Neurogene’s business following the merger. Such litigation may have an adverse impact on the combined company’s business and results of operations or may cause disruptions to the combined company’s operations. In addition, in the past, stockholders have initiated class action lawsuits against biotechnology companies following periods of volatility in the market prices of these companies’ stock. Such litigation, if instituted against the combined company, could cause the combined company to incur substantial costs and divert management’s attention and resources, which could have a material adverse effect on the combined company’s business, financial condition and results of operations.

If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about the combined company, its business or its market, its stock price and trading volume could decline.

The trading market for the combined company’s common stock will be influenced by the research and reports that equity research analysts publish about it and its business. Equity research analysts may elect to not provide research coverage of the combined company’s common stock after the completion of the merger, and such lack of research coverage may adversely affect the market price of its common stock. In the event it does

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have equity research analyst coverage, the combined company will not have any control over the analysts or the content and opinions included in their reports. The price of the combined company’s common stock could decline if one or more equity research analysts downgrade its stock or issue other unfavorable commentary or research. If one or more equity research analysts ceases coverage of the combined company or fails to publish reports on it regularly, demand for its common stock could decrease, which in turn could cause its stock price or trading volume to decline.

The combined company will have broad discretion in the use of the cash and cash equivalents of the combined company and the proceeds from the Neurogene pre-closing financing and may invest or spend the proceeds in ways with which you do not agree and in ways that may not increase the value of your investment.

The combined company will have broad discretion over the use of the cash and cash equivalents of the combined company and the proceeds from the Neurogene pre-closing financing. You may not agree with the combined company’s decisions, and its use of the proceeds may not yield any return on your investment. The combined company’s failure to apply these resources effectively could compromise its ability to pursue its growth strategy and the combined company might not be able to yield a significant return, if any, on its investment of these net proceeds. You will not have the opportunity to influence its decisions on how to use the combined company’s cash resources.

The combined company may be subject to adverse legislative or regulatory tax changes that could negatively impact its financial condition.

The rules dealing with U.S. federal, state and local income taxation are constantly under review by persons involved in the legislative process and by the Internal Revenue Service and the U.S. Treasury Department. Changes to tax laws (which changes may have retroactive application) could adversely affect the combined company or its stockholders. The combined company will assess the impact of various tax reform proposals and modifications to existing tax treaties in all jurisdictions where the combined company has operations to determine the potential effect on its business and any assumptions the combined company will make about its future taxable income. It cannot predict whether any specific proposals will be enacted, the terms of any such proposals or what effect, if any, such proposals would have on its business if they were to be enacted. For example, the United States recently enacted the Inflation Reduction Act of 2022, which implements, among other changes, a 1% excise tax on certain stock buybacks. In addition, beginning in 2022, the Tax Cuts and Jobs Act eliminated the option to deduct research and development expenditures and requires taxpayers to amortize them generally over five years. The U.S. Congress is considering legislation that would restore the current deductibility of research and development expenditures; however, there is no assurance that the current provision will be repealed or otherwise modified. Such changes, among others, may adversely affect the combined company’s effective tax rate, results of operation and general business condition.

The combined company’s ability to use net operating loss carryforwards and other tax attributes may be limited, including as a result of the merger.

Under current law, U.S. federal net operating loss carryforwards generated in taxable periods beginning after December 31, 2017, may be carried forward indefinitely, but the deductibility of such net operating loss carryforwards is limited to 80% of taxable income. In addition, under Sections 382 and 383 of the Code, U.S. federal net operating loss carryforwards and other tax attributes may become subject to an annual limitation in the event of certain cumulative changes in ownership. An “ownership change” pursuant to Section 382 of the Code generally occurs if one or more stockholders or groups of stockholders who own at least 5% of a company’s stock increase their ownership by more than 50 percentage points over their lowest ownership percentage within a rolling three-year period. The combined company’s ability to utilize its net operating loss carryforwards and other tax attributes to offset future taxable income or tax liabilities may be limited as a result of ownership changes, including, as discussed above, in connection with the merger or other transactions. Similar rules may apply under state tax laws. If the combined company earns taxable income, such limitations could result in increased future income tax liability to the combined company, and the combined company’s future cash flows could be adversely affected.

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Unfavorable global economic conditions could adversely affect the combined company’s business, financial condition, results of operations or cash flows.

The combined company’s results of operations could be adversely affected by general conditions in the global economy and in the global financial markets. A severe or prolonged economic downturn could result in a variety of risks to the combined company’s business, including weakened demand for the combined company’s product candidates and the combined company’s ability to raise additional capital when needed on acceptable terms, if at all. A weak or declining economy could also strain the combined company’s suppliers, possibly resulting in supply disruption, or cause the combined company’s customers to delay making payments for its services. Any of the foregoing could harm the combined company’s business and the combined company cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact its business.

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MARKET PRICE AND DIVIDEND INFORMATION

The closing price of the Neoleukin common stock on July 17, 2023, the last day of trading prior to the announcement of the merger, as reported on The Nasdaq Capital Market, was $0.925 per share, or $4.625 after giving effect to the 1-for-5 reverse stock split that was effected on September 25, 2023. The closing price of the Neoleukin common stock on      , 2023, the last practicable date before the date of this proxy statement/prospectus, as reported on Nasdaq, was $    per share.

Because the market price of the Neoleukin common stock is subject to fluctuation, the market value of the shares of the Neoleukin common stock that the Neurogene stockholders will be entitled to receive in the merger may increase or decrease.

Assuming approval of Proposal No. 1 and at least one of Proposal Nos. 2 or 4 and successful application for initial listing with Nasdaq, following the consummation of the merger, the Neoleukin common stock will trade on Nasdaq under Neoleukin’s new name, “Neurogene Inc.,” and new trading symbol “NGNE.”

As of     , 2023, the record date for the special meeting, there were approximately    registered holders of record of the Neoleukin common stock. As of     , 2023, Neurogene had    holders of record of Neurogene common stock and    holders of record of Neurogene preferred stock. For detailed information regarding the beneficial ownership of certain Neoleukin and Neurogene stockholders, see the sections of this proxy statement/prospectus entitled “Principal Stockholders of Neoleukin” and “Principal Stockholders of Neurogene.”

Dividends

Neoleukin has never declared or paid any cash dividends on the Neoleukin common stock and does not anticipate paying cash dividends on the Neoleukin common stock for the foreseeable future, except pursuant to the CVR Agreement if the combined company’s board of directors and management elects to make such payments, if any, in cash instead of stock. Notwithstanding the foregoing, any determination to pay cash dividends subsequent to the merger will be at the discretion of the combined organization’s then-current board of directors and will depend upon a number of factors, including the combined organization’s results of operations, financial condition, future prospects, contractual restrictions, restrictions imposed by applicable law and other factors the then-current board of directors deems relevant.

Neurogene has never paid or declared any cash dividends on the Neurogene capital stock. If the merger does not occur, Neurogene does not anticipate paying any cash dividends on the Neurogene capital stock in the foreseeable future, and Neurogene intends to retain all available funds and any future earnings to fund the development and expansion of its business. Any future determination to pay dividends on Neurogene stock if the merger is not completed will be at the discretion of the Neurogene board of directors and will depend upon a number of factors, including its results of operations, financial condition, future prospects, contractual restrictions, and restrictions imposed by applicable laws and other factors the Neurogene board of directors deems relevant.

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CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This proxy statement/prospectus contains or incorporates statements that constitute forward-looking statements within the meaning of the federal securities laws in relation to Neoleukin, Neurogene, the merger and the other proposed transactions contemplated thereby. Any express or implied statements that do not relate to historical or current facts or matters are forward-looking statements. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. These forward-looking statements include, but are not limited to, express or implied statements regarding Neoleukin’s or Neurogene’s expectations, hopes, beliefs, intentions or strategies regarding the future. In some cases, you can identify forward-looking statements by terminology such as “may,” “might,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “target,” “endeavor,” “possible,” “potential,” “continue,” “contemplate” or the negative of these terms or other comparable terminology, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements are based on current expectations and beliefs concerning future developments and their potential effects. There can be no assurance that future developments affecting Neoleukin, Neurogene or the proposed transaction will be those that have been anticipated. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Neoleukin’s or Neurogene’s control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. In addition to other factors and matters contained in or incorporated by reference in this document, Neoleukin and Neurogene believe the following factors could cause actual results to differ materially from those discussed in the forward-looking statements:

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