Neurogene Inc.

Neurogene Inc. is a clinical-stage biotechnology company dedicated to developing life-changing genetic medicines for rare and devastating neurological diseases. The company's core mission is to transform these complex conditions into treatable ones, thereby improving the lives of affected patients and their families. Neurogene's corporate headquarters are located in New York, NY.

Neurogene leverages its proprietary EXACT™ (Expression Attenuation via Construct Tuning) transgene regulation technology, designed to deliver therapeutic protein levels with precise control and minimize potential toxicity associated with conventional gene therapy. The company's pipeline includes NGN-401, an investigational AAV9 gene therapy for Rett syndrome, which is currently in Phase 1/2 clinical trials and has received FDA Breakthrough Therapy designation. Additionally, Neurogene is developing NGN-101, a conventional gene therapy candidate for CLN5 Batten disease, also in Phase 1/2 clinical trials.

Recent notable developments for Neurogene include the pricing of a $125 million public offering in July 2026. The company also announced the successful completion of dosing in the Embolden™ registrational trial for NGN-401 for Rett syndrome. Rachel McMinn, Ph.D., serves as the Founder and Chief Executive Officer, leading the company's strategic direction. Neurogene also operates a manufacturing facility in Houston, Texas, supporting its clinical development activities.