Neurogene
Neurogene develops genetic medicines for neurological diseases, focusing on rare conditions like Rett syndrome and CLN5 Batten disease.
Company Overview
Neurogene focuses on developing genetic medicines aimed at transforming the lives of individuals and families affected by severe neurological diseases. The company is building a gene therapy pipeline to address the high unmet needs for rare neurological diseases. Neurogene's corporate headquarters are located at 535 W 24th Street, 5th Floor, New York, NY 10011, with manufacturing headquarters in Houston, TX 77025.
Gene Therapy Pipeline
Neurogene is at the forefront of creating gene therapies for rare neurological diseases. They presented favorable safety data from their Phase 1/2 NGN-401 gene therapy trial for Rett syndrome at the ASGCT Annual Meeting. NGN-401 is an investigational AAV9 gene therapy intended as a one-time treatment for Rett syndrome, while NGN-101 is a development candidate for the treatment of CLN5 Batten disease. Their pipeline addresses significant unmet medical needs in these areas.
EXACT Gene Therapy Technology
Neurogene is developing the EXACT gene therapy technology to revolutionize treatment possibilities for complex genetic diseases. EXACT technology aims to deliver highly controlled and consistent levels of transgene expression. This approach is instrumental in achieving accurate therapeutic outcomes for patients with severe neurological conditions.
Manufacturing and Platform Processes
Neurogene has developed two robust and scalable platform processes to support the production of research and clinical grade material. These processes utilize either HEK293 (mammalian) or Sf9 (insect) cells in suspension. Their 42,000 square foot facility employs single-use technologies, enabling the production of multiple products under one roof. This facility is located in Houston, TX 77025.
Research Collaborations
Neurogene is engaged in a research collaboration with the University of Edinburgh. This partnership focuses on biomedical and translational research for neurodevelopmental diseases. The collaboration underscores Neurogene's commitment to integrating cutting-edge research and the perspectives of the rare patient community into their innovative therapeutic approaches.