A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
Gene therapy clinical trials are research studies that help determine whether a gene therapy is safe and effective to treat a specific genetic disease.
Transgene Regulation
CNS + Ocular Delivery
Product
Candidate
Candidate
Indication
Discovery
IND* Enabling
Phase I/2
Near-Term
Expected Milestones
Expected Milestones
Early Discovery
Undisclosed
Initiate Clinical Study
2025
2025
*IND = investigational new drug
Partnering with the University of Edinburgh
Neurogene has a research collaboration with the University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases.
The collaboration provides comprehensive research capabilities to Neurogene, enabling the company and the University to accelerate scientific innovation to continue to improve upon conventional gene therapy.
Clinical Trials
Gene Therapy Study for Children With CLN5 Batten Disease
(CLN5-200)
Gene therapy clinical trials are research studies that help determine whether a gene therapy is safe and effective to treat a specific genetic disease
A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)
A natural history study is an essential component of developing and expediting treatments, where the natural history is not well documented. Participating in a natural history study enables a rigorous scientific evaluation of disease characteristics and disease progression, which provides essential information to design a clinical trial that allows for the evaluation of whether a treatment is safe and effective.
