Neoleukin Therapeutics

Neurogene, Inc. is a clinical-stage biotechnology company, which engages in the development of product portfolio of genetic medicines for rare neurological diseases. The company is headquartered in New York City, New York and currently employs 107 full-time employees. The company went IPO on 2014-03-07. 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 in 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 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. The company is also developing NGN-101 for the treatment of CLN5 Batten disease.