Neurogene Inc.

Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. Its primary purpose is to create purpose-built gene therapies using a biology-first approach, optimizing gene selection, construct design, expression control, and delivery to target neural systems effectively while prioritizing safety. The lead program, NGN-401, is an investigational AAV9 gene therapy delivering the full-length human MECP2 gene under EXACT transgene regulation technology for Rett syndrome, currently in the ongoing Embolden registrational trial evaluating a single dose in females aged three and older. This program has received multiple FDA designations including Breakthrough Therapy, RMAT, Orphan Drug, Fast Track, and Rare Pediatric Disease, along with similar recognitions from EMA and UK MHRA. Neurogene also advances NGN-101 for CLN5 Batten disease, with Phase 1/2 trial enrollment completed and follow-up ongoing. Founded in 2018 and headquartered in New York, NY, Neurogene addresses high unmet needs in rare neurological disorders through innovative gene therapy platforms.