Neurogene Inc.

Neurogene Inc. (NASDAQ: NGNE) is a clinical-stage biotechnology company dedicated to developing genetic medicines for rare neurological diseases with high unmet medical need. The company's website is www.neurogene.com.

Founded in January 2018, Neurogene focuses on overcoming the limitations of conventional gene therapy through its proprietary EXACT (Expression Attenuation via Construct Tuning) platform technology. EXACT utilizes microRNA-based genetic circuits designed to deliver therapeutic levels of transgene to key brain areas while maintaining precise control over expression levels. This approach addresses dosage-sensitive diseases with narrow therapeutic windows that were previously considered untreatable with standard gene therapy approaches.

The company's lead program, NGN-401, is in Phase 3 clinical development for Rett syndrome, a severe X-linked neurodevelopmental disorder caused by mutations in the MECP2 gene. NGN-401 contains the full-length MECP2 gene and is administered via intracerebroventricular infusion to achieve optimal biodistribution across brain regions relevant to disease pathophysiology. The registrational Embolden trial is a single-arm, open-label study evaluating NGN-401 in 20 females ages three and above, with enrollment complete and dosing more than 50 percent complete as of the filing date.

Neurogene operates a fully functional current good manufacturing practices facility in Houston, Texas, spanning approximately 42,000 square feet with 6,000 square feet of dedicated cleanroom space. This in-house manufacturing capability enables the company to maintain control over product quality, development timelines, and strategic pipeline flexibility while supporting the transition from preclinical through commercial-stage manufacturing.

As of March 16, 2026, the company employed 131 full-time employees, with 106 engaged in research, development, and technical operations. Thirty employees hold Ph.D., M.D., D.O., or Pharm.D. degrees. The company has established collaborations with the University of Edinburgh to support research and pipeline development activities. NGN-401 has received Breakthrough Therapy designation from the FDA, Priority Medicines designation from the European Medicines Agency, and Regenerative Medicine Advanced Therapy designation, positioning it for expedited development and review pathways.