Posted by Michael Wonder on 27 Feb 2026
Neurogene announces FDA breakthrough therapy designation for NGN-401 gene therapy for Rett syndrome
26 February 2026 - Breakthrough therapy designation granted based on interim NGN-401 Phase 1/2 data demonstrating clinically meaningful, durable and multi-domain functional improvements.
Neurogene today announced that the US FDA has granted breakthrough therapy designation to NGN-401, an investigational gene therapy in late stage clinical development as a potential best-in-class treatment for Rett syndrome.
