Neurogene reports positive Phase 1/2 data for NGN-401 in Rett syndrome; 47 milestones gained across 10 patients

Neurogene Inc.

47 developmental milestones gained across 10 participants (avg 4.7); 100% improved CGI-I and gained ≥1 milestone.
Durable effect through 30 months with no plateau or milestone loss; median time to first improvement 2 months.
NGN-401 generally well-tolerated; no new treatment-related SAEs since Oct 2025; all AEs mild or moderate.
Dosing complete in Embolden registrational trial; no treatment-related SAEs or DLTs; topline data expected 2H 2027.
Improvements across RSGMS and RSHFS scales (p<0.001); 7/10 gained ≥2 milestones across ≥2 Rett domains.

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