Neurogene, Inc. (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on discovering, developing and commercializing next-generation gene therapies for patients suffering from rare neurological disorders. Utilizing proprietary adeno-associated virus (AAV) vectors optimized for central nervous system delivery, the company’s research platform aims to address unmet medical needs in both monogenic and complex neurodegenerative diseases. Neurogene’s pipeline includes multiple in-house programs targeting inherited pediatric disorders, adult-onset movement disorders and other central nervous system indications.
Among its lead candidates is a program designed to deliver disease-modifying transgenes directly into the brain via convection-enhanced delivery, with initial trials in Parkinson’s disease. In parallel, preclinical assets are advancing toward first-in-human studies in frontotemporal dementia and amyotrophic lateral sclerosis (ALS). Neurogene’s approach emphasizes long-lasting therapeutic benefit from a single administration, leveraging collaborations with academic research centers and specialized contract manufacturing organizations to support scalable vector production and regulatory filings.
Established in 2019 and headquartered in Cambridge, Massachusetts, Neurogene operates a state-of-the-art cGMP manufacturing facility for AAV vector production, alongside research laboratories dedicated to vector engineering and in vivo neuroscience models. The company’s leadership team brings deep expertise from leading gene therapy and neuroscience organizations, including roles in late-stage clinical development, regulatory affairs and biomanufacturing. This collective experience supports a strategic focus on stringent safety assessment and robust clinical trial design.
Neurogene maintains strategic partnerships and licensing arrangements in North America, Europe and Asia to accelerate global development of its pipeline candidates. By combining innovative vector technology with a targeted approach to central nervous system delivery, the company aims to transform the treatment landscape for patients with debilitating neurological conditions, ultimately delivering durable therapeutic solutions where few options currently exist.
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