Neurogene Inc.

Neurogene is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases using its proprietary EXACT platform, which employs microRNA-based genetic circuits to achieve precise therapeutic transgene expression in the brain. The company's lead program, NGN-401, is designed to treat Rett syndrome, a severe X-linked neurodevelopmental disorder caused by MECP2 gene mutations, using a one-time gene therapy delivery to address the disease's narrow therapeutic window. Neurogene generates no revenue currently and operates on equity financing and grant support, with a business model dependent on successful clinical development and eventual commercialization of its product candidates; the company also operates an internal cGMP manufacturing facility to support its pipeline development and future commercial production.