NEUROGENE INC (NGNE)
Health Care · Gene therapy / genetic medicines for neurological diseases · NASDAQ
Early-stage gene therapy company using proprietary microRNA-circuit technology to deliver full-length MECP2 gene therapy for Rett syndrome via brain injection.
What NEUROGENE INC does
Neurogene is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases. The company leverages its proprietary EXACT™ (Expression Attenuation via Construct Tuning) transgene regulation technology, which uses microRNA-based genetic circuits to deliver therapeutic levels of transgene to key brain areas. Its lead program, NGN-401, is in registrational trial (Embolden) for Rett syndrome using AAV delivery via intracerebroventricular administration, with an in-house cGMP manufacturing facility in Houston, Texas.
Themes: ["Gene therapy / genetic medicines","Rare neurological diseases","Rett syndrome","AAV therapeutics","CNS / neurological drug development"]
Fundamentals
- Price$42.84 as of 2026-07-09 close
- Market cap$843M as of 2026-07-10
- 1-year return+101.5% as of 2026-07-09 close
- Net margin-8123.7% as of 2026-07-10
- ROE-25.3% as of 2026-07-10
- Debt / equity0.00 as of 2026-07-10
- Beta1.67 as of 2026-07-10
Key risks (from latest filing)
["Clinical development risk: NGN-401 is in early registrational trial for Rett syndrome with uncertain regulatory approval pathway; failure to meet efficacy endpoints would block commercialization","Capital requirements: Company is pre-commercial with accumulated deficit of $352.6 million and net losses of $90.4 million in 2025; requires additional financing to fund ongoing clinical development and manufacturing scale-up","Manufacturing and supply chain: Reliance on in-house cGMP facility and AAV manufacturing capacity to support clinical trials and future commercial scale-up; any manufacturing disruption could delay programs"]
Competitors & peers
- Ultragenyx Pharmaceutical (RARE)
- Regenxbio (RGNX)
- Solid Therapeutics (SLDT)
- Audentes Therapeutics (now part of Astellas)
- Spark Therapeutics (ONCE, now part of Roche)
Fundamentals: Finnhub, as of 2026-07-10. Filings: SEC EDGAR. Prices are delayed daily-close data.
Last updated 2026-07-09.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.