• Neurogene highlighted NGN-401 as a one-time gene therapy candidate for Rett syndrome, positioned as a potential best-in-class program.
• Phase 1/2 update: 100% of 10 participants gained at least 1 developmental milestone and improved on CGI-I at 12 months or longer.
• Durability signal: continued improvement reported through 30 months post-dose, with no milestone losses; 80% met the Embolden composite responder definition at 12 months.
• Embolden registrational trial completed dosing of 25 participants, exceeding target enrollment; topline data expected in 2H 2027.
• Manufacturing milestone: completion of BLA-enabling process performance qualification runs expected by year-end 2026.

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 on August 11, 2026, and is solely responsible for the information contained therein.