OUR PIPELINE
We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
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 >>We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
At Neurogene, we are driven by a strong sense of purpose to advance genetic medicines.
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 MoreNeurogene 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.