NGN-401 gene therapy for Rett syndrome received RMAT designation from FDA based on preliminary clinical evidence indicating the potential to address unmet medical needs NGN-401 selected for FDA START Program, also designed to accelerate development Interim NGN-401 efficacy data from Cohort 1
Designation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet medical needs in Rett syndrome RMAT designation provides an opportunity for an Accelerated Approval pathway under the 21st Century Cures Act, and is in addition to NGN-401’s
NEW YORK --(BUSINESS WIRE)--Jul. 2, 2024-- Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the BMO 2024 Genetic
NEW YORK --(BUSINESS WIRE)--Jul. 1, 2024-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Neurogene will be added to the Russell 3000 ® Index, effective at
High-dose NGN-401 has been well-tolerated, and low-dose NGN-401 continues to show a favorable safety profile Interim safety data presented at the International Rett Syndrome Foundation (IRSF) ASCEND Summit NEW YORK --(BUSINESS WIRE)--Jun. 18, 2024-- Neurogene Inc.
NEW YORK --(BUSINESS WIRE)--Jun. 5, 2024-- Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following conferences:
NGN-401 is one of only three CBER programs chosen by FDA NGN-401 was selected based on potential for clinical benefits and clinical development program readiness START Program provides sponsors enhanced communications with FDA to accelerate development of rare disease therapies NEW YORK --(BUSINESS
Presented favorable safety data from Phase 1/2 NGN-401 gene therapy trial for Rett syndrome at ASGCT Annual Meeting Received Australian HREC approval for NGN-401 trial Remains on track to provide interim NGN-401 efficacy data from Cohort 1 in 4Q:24 Strong balance sheet with cash runway into 2H:26
NGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-up No signs or symptoms of overexpression toxicity, including in one patient with a mild variant predicted to result in residual MeCP2 expression Neurogene remains on track to provide
NGN-401 has been generally well-tolerated by three patients with multiple months of follow-up NEW YORK --(BUSINESS WIRE)--Apr. 22, 2024-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological
