Neurogene reports durable Phase 1/2 NGN-401 gains: 47 milestones across 10 patients, Embolden topline due 2H 2027

Neurogene reported positive Phase 1/2 results for NGN-401 showing 47 developmental milestones gained across 10 participants and durable improvement through 30 months.Key Highlights:Original SEC Filing: Neurogene Inc. [ NGNE ] - 8-K - Jun. 29, 2026This is an AI-powered summary. It may contain inaccu…

Neurogene reported positive Phase 1/2 results for NGN-401 showing 47 developmental milestones gained across 10 participants and durable improvement through 30 months.

Key Highlights:

47 total developmental milestones gained across 10 participants (4.7 avg per participant), indicating multidomain functional gains.
100% of participants improved on CGI-I and gained ≥1 milestone; all gained ≥1 milestone in past 12 months.
Milestone gains were progressive and durable through 30 months with no plateau or loss observed.
NGN-401 1E15 vg dose generally well-tolerated; no new treatment-related SAEs or DLTs in Embolden as of June 16, 2026.
Embolden registrational trial dosing complete (N=25); topline data expected in 2H 2027, supporting potential BLA path.

Original SEC Filing:

This is an AI-powered summary. It may contain inaccuracies. Consider verifying important information with the source. Please note this summary is solely based on documents filed with the SEC.