Neurogene FDA agreement on Rett gene therapy registrational trial; Q4 2025 dosing start

Neurogene Inc.

Completed FDA discussions on Embolden registrational trial protocol for NGN-401; first participant dosing planned in Q4 2025 across 13 U.S. sites.
New preclinical NHP data shows ICV delivery achieves superior brain biodistribution vs. IT-L at clinically relevant 1E15 vg dose; supports ICV route.
Phase 1/2 data: first 4 participants gained 23 developmental milestones across multiple domains, indicating multi-domain improvement.
ICV and IT-L show comparable peripheral organ transduction; no liver-sparing benefit to IT-L administration.

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