Neurogene Inc. reported a net loss of $90.4 million for the year ended December 31, 2025, compared to a net loss of $75.1 million for the previous year. The company's operating expenses totaled $103.3 million in 2025, up from $83.5 million in 2024, with research and development expenses accounting for $75.0 million and $60.9 million, respectively. The increase in research and development expenses was primarily driven by the advancement of the NGN-401 program, including clinical trial costs and manufacturing expenses. Revenue under licensing agreements was $0 for 2025, compared to $0.9 million in 2024, which was generated from the recognition of upfront payments related to legacy Neoleukin assets.
The company's strategic focus remains on the clinical development of NGN-401 for Rett syndrome, with the Embolden registrational trial fully enrolled and dosing expected to complete in the second quarter of 2026. Neurogene is also advancing early-stage discovery programs leveraging its EXACT platform for other neurological diseases. However, the company decided not to move forward with the NGN-101 program for CLN5 Batten disease due to the lack of a streamlined regulatory pathway. Neurogene's team has grown to 131 employees as of March 16, 2026, with 106 engaged in research, development, and technical operations.
Key operational developments include the FDA's acceptance of NGN-401 into the START Pilot Program and the granting of Breakthrough Therapy and RMAT designations. These designations are intended to expedite the development and review process. The company also received PRIME designation from the EMA. Neurogene's in-house cGMP manufacturing facility in Houston, Texas, is fully operational and supports the production of clinical-grade NGN-401 for ongoing trials. The company believes that its in-house manufacturing capabilities enable better control of product quality, development timelines, and strategic pipeline flexibility.
Looking ahead, Neurogene anticipates that its expenses and capital requirements will increase substantially as it continues to advance the NGN-401 program, invest in research programs, and expand its clinical, scientific, management, and administrative teams. Based on its current operating plan, the company believes that its existing cash, cash equivalents, and short-term investments should be sufficient to fund operations through the first quarter of 2028. However, the company acknowledges that it will require substantial additional capital to finance its operations in the future and that there is no assurance that it will be able to raise such capital on acceptable terms, or at all.
About Neurogene Inc.
Neurogene Inc. is a biotechnology company focused on developing gene therapies for rare, complex neurological diseases. Utilizing proprietary EXACT transgene regulation technology and in-house manufacturing, it advances clinical-stage programs like NGN-401 for Rett syndrome and pipeline candidates for other neurodegenerative disorders. The company leverages collaborations with academic institutions, targeting unmet medical needs with innovative, scalable genetic medicines delivered via optimized routes to the central nervous system.
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