NGNE

Neurogene Inc. is a clinical-stage biotechnology company. The Company, through its proprietary transgene regulation technology, Expression Attenuation via Construct Tuning (EXACT), is building a product portfolio of genetic medicines for rare neurological diseases with high unmet needs not otherwise addressable by conventional gene therapy. Its clinical-stage program, NGN-401, utilize the EXACT platform, which is under development for the treatment of Rett syndrome, a disease with a patient population that has a significant unmet need, and that ultimately progresses to substantial neurological and physical impairment and premature death. NGN-401 is being evaluated in pediatric female patients with Rett syndrome in a Phase 1/2 open-label, multi-center clinical trial that assesses the safety, tolerability, and efficacy of two doses of NGN-401 delivered using a one-time intracerebral ventricular (ICV) procedure. It is also developing NGN-101 for the treatment of CLN5 Batten disease.