Neurogene

Neurogene is dedicated to developing life-changing genetic medicines for individuals and families affected by severe neurological diseases. The company is building a gene therapy pipeline to address the high unmet need for rare neurological conditions. Neurogene’s corporate headquarters are located at 535 W 24th Street, 5th Floor, New York, NY 10011, with manufacturing headquarters in Houston, TX 77025. They are developing the EXACT gene therapy technology, which delivers highly controlled and consistent levels of transgene expression, to reimagine treatment possibilities for complex genetic diseases. Neurogene has established two robust and scalable platform processes for producing research and clinical grade material using HEK293 (mammalian) or Sf9 (insect) cells in suspension, and employs single-use technologies to allow multiple products to be produced at their 42,000 square foot facility. The company has partnered with the University of Edinburgh for research collaboration in biomedical and translational research for neurodevelopmental diseases. Neurogene’s investigational AAV9 gene therapy, NGN-401, is being developed as a one-time treatment for Rett syndrome, and NGN-101 is a development candidate for the treatment of CLN5 Batten disease. The company has presented favorable safety data from its Phase 1/2 NGN-401 gene therapy trial for Rett syndrome at the ASGCT Annual Meeting. Neurogene is committed to integrating the perspective of the rare patient community into their work and currently does not offer an expanded access program for investigational gene therapies outside of clinical trials. Their clinical trials include studies for NGN-401 gene therapy for female children with Rett syndrome and a gene therapy study for children with CLN5 Batten Disease.