Neurogene doses first participant in Embolden registrational trial of NGN-401 for Rett syndrome

Neurogene Inc.

First participant dosed in single-arm, open-label, baseline-controlled registrational trial of NGN-401 gene therapy for Rett syndrome.
Trial targets 20 females ages 3+; enrollment expected to complete in 3-6 months.
12 of 13 clinical sites initiated; trial designed to support future BLA submission.
Company to present Phase 1/2 interim data at Stifel Healthcare Conference on November 12, 2025.

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.