Neurogene Inc. is a clinical-stage biotechnology company dedicated to developing life-changing genetic medicines for patients and families affected by rare neurological diseases. The company's core innovation is its proprietary EXACT (Expression Attenuation via Construct Tuning) transgene regulation platform. This technology is designed to overcome the limitations of conventional gene therapy by delivering therapeutic levels of a transgene within a narrow, targeted range, thereby avoiding the toxicities associated with transgene overexpression.
Neurogene's lead product candidate is NGN-401, an investigational adeno-associated virus (AAV9) gene therapy currently in Phase 1/2 clinical trials for the treatment of Rett syndrome. The company has also developed NGN-101, a gene therapy candidate for the treatment of CLN5 Batten disease, alongside multiple discovery-stage candidates targeting other central nervous system disorders. As a pre-revenue clinical-stage company, Neurogene does not currently generate product revenue and funds its operations through equity financings, including a reverse merger with Neoleukin Therapeutics in December 2023 and subsequent private investments. The company operates primarily out of its headquarters in New York and maintains internal manufacturing capabilities to support its clinical development and future commercialization efforts.
