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.
Learn More >>
ABOUT US
At Neurogene, we are driven by a strong sense of purpose to advance genetic medicines.
LATEST NEWS
Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome
Two pediatric patients with Rett syndrome dosed in the United States with NGN-401, Neurogene’s lead gene therapy product candidate leveraging…
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.