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OUR PIPELINE
We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
Focusing on the Future of Life-Changing
Genetic Medicines
Gene therapy holds incredible promise to revolutionize medical treatment. At Neurogene we believe that innovation, robust scientific execution, and a strong sense of purpose are required to turn this promise into reality. Our focus is to develop life-changing genetic medicines for people and their families impacted by devastating neurological diseases.
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ABOUT US
At Neurogene, we are driven by a strong sense of purpose to advance genetic medicines.
LATEST NEWS
Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome
Planned NGN-401 Phase 1/2 clinical trial to dose female pediatric patients with Rett syndrome in 2023 In a comprehensive preclinical…
Learn MoreState of the Art Manufacturing
Neurogene owns and operates a state-of-the-art gene therapy manufacturing facility in Houston, Texas with fully integrated process and analytical development, and CGMP capabilities. We have developed two scalable AAV manufacturing processes (mammalian and insect cell-based production) to support preclinical studies and clinical trials.