Neoleukin Therapeutics Inc

Neurogene Inc. (Nasdaq: NGNE) is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases. The company employs a biology-first approach paired with optimized delivery to develop purpose-built gene therapies, including programs powered by its proprietary EXACT transgene regulation technology for precise transgene expression. Its lead program, NGN-401, is an investigational AAV9 gene therapy in late-stage clinical development as a potential one-time treatment for Rett syndrome — a devastating neurological disorder caused by mutations in the MECP2 gene affecting approximately 15,000–20,000 patients in the US, EU, and UK. NGN-401 is the only clinical candidate designed to deliver the full-length human MECP2 gene via intracerebroventricular administration for broad brain and nervous system targeting. The company operates a wholly owned, fully integrated in-house AAV manufacturing facility in Houston. NGN-401 has received multiple regulatory designations including FDA Breakthrough Therapy, RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, as well as EMA PRIME and ATMP designations. The company generates no product revenue and is funded through equity offerings. Updated Aug 11, 2026