Neurogene presents NGN-401 data showing developmental milestone gains in all 10 Phase 1/2 participants
NGNE•Neurogene said all 10 participants in its Phase 1/2 NGN-401 trial gained developmental milestones, with no losses observed through 30 months of follow-up. It completed dosing 25 participants in its Embolden registrational trial, 25% above its enrollment target, and expects topline results in the second half of 2027.
1. Phase 1/2 results
All 10 participants gained developmental milestones, and no milestone losses were observed through 30 months of follow-up. At 12 months, 80% met Embolden’s composite responder definition.
2. Embolden trial update
Neurogene completed dosing 25 participants in its Embolden registrational trial of NGN-401 for Rett syndrome, exceeding its enrollment target by 25%. Topline results are expected in the second half of 2027. Completion of BLA-enabling Process Performance Qualification runs is expected by year-end 2026.
