Neurogene Inc announces Embolden trial for NGN401 gene therapy in Rett syndrome to begin dosing in Q4 2025

Reuters

Oct 09

Neurogene Inc announces Embolden trial for NGN401 gene therapy in Rett syndrome to begin dosing in Q4 2025

Neurogene Inc., a clinical-stage company focused on genetic medicines for rare neurological diseases, announced the completion of discussions with the U.S. Food and Drug Administration (FDA) regarding the protocol for its Embolden™ registrational trial of NGN-401 gene therapy in Rett syndrome. The company plans to initiate dosing in the fourth quarter of 2025, with the trial enrolling participants across 13 sites. Neurogene also reported new preclinical data from nonhuman primates demonstrating that intracerebroventricular (ICV) delivery of NGN-401 achieves superior AAV biodistribution in brain regions relevant to Rett syndrome compared to intrathecal lumbar (IT-L) delivery. These results will be presented on October 9, 2025, and will be available on Neurogene's website.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251009995946) on October 09, 2025, and is solely responsible for the information contained therein.

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