Neurogene Inc. (NASDAQ: NGNE) is a clinical-stage gene therapy company developing treatments for rare neurological diseases. The company generates no product revenue, funding operations entirely through capital raises while spending on research and development. Its lead program, NGN-401, targets Rett syndrome using its proprietary EXACT technology delivered via adeno-associated virus (AAV) in a one-time intracerebroventricular procedure. The registrational Embolden trial, a single-arm, open-label, baseline-controlled study enrolling 20 females with Rett syndrome, was 100% enrolled as of the FY2025 10-K filing date, with more than 50% of participants dosed. Total research and development expenses were $75.0 million in FY2025, up from $60.9 million in FY2024, with Rett syndrome program costs alone reaching $25.8 million in FY2025. A Batten disease program was de-prioritized during FY2025. Rachel McMinn, Ph.D. serves as Chief Executive Officer.

Revenue model

Neurogene has no approved products and no product revenue as of the FY2025 10-K filing. Operations are funded through capital markets. All expenditures are research and development costs, including payments to contract research organizations, contract development and manufacturing organizations, and internal manufacturing scale-up.

Products and services

NGN-401: gene therapy for Rett syndrome using EXACT technology and AAV delivery via one-time intracerebroventricular administration, currently in the Embolden registrational trial (FY2025). A Batten disease program was de-prioritized in FY2025. Early discovery programs also exist but were reduced in spending from $5.4 million in FY2024 to $3.2 million in FY2025.

Customers and end markets

Target patient population is females with Rett syndrome, a rare progressive neurodevelopmental disease. The Embolden trial enrolls females age three and above. No commercial customers exist as of the FY2025 10-K filing date.

Value-chain role

Drug developer and gene therapy manufacturer. Neurogene operates its own current good manufacturing practices facility for clinical-grade NGN-401 and contracts with third-party CROs and CDMOs for clinical trials and additional manufacturing. The company has no commercial sales or distribution operations.

Geographic exposure

Primary operations in the United States. Subject to international regulatory requirements for potential future markets outside the U.S. Relies to some degree on foreign CROs and CDMOs, with exposure to trade restrictions and tariffs affecting foreign-sourced materials, including from China.

Source: SEC 10-K, filed 2026-03-24

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