Neurogene publishes corporate presentation highlighting NGN-401 gene therapy program for Rett syndrome

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.