Neurogene Inc.

Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases that are not treatable by conventional gene therapy. The company aims to address the challenges of complex neurological disease biology and variable gene uptake and expression. Neurogene Inc. utilizes its proprietary transgene regulation technology, EXACT™ (Expression Attenuation via Construct Tuning), which employs microRNA-based genetic circuits. This technology is designed to deliver therapeutic levels of transgenes to specific brain areas involved in neurological disease pathology. Neurogene Inc.'s primary clinical-stage program is NGN-401, which is being developed for the treatment of Rett syndrome. NGN-401 uses the EXACT technology and adeno-associated virus (AAV) delivery. Rett syndrome is a severe and progressive neurodevelopmental disease causing significant neurological and physical impairment. The company's ongoing registrational trial for NGN-401, named Embolden™, is a single-arm, open-label, baseline-controlled trial. This trial is evaluating the 1E15 vg dose of NGN-401 in 20 females with Rett syndrome, aged three and above. Embolden has enrolled 100% of participants, and more than 50% of participants have been dosed. Dosing is expected to be completed in the second quarter of 2026. Neurogene Inc. also completed dosing in a Phase 1/2 open-label, multi-center clinical trial of NGN-401 for Rett syndrome, where ten participants received the 1E15 vg dose. NGN-401 is administered via a one-time intracerebroventricular (ICV) procedure.