*EXACT = Expression Attenuation via Construct Tuning
**POC = Proof of Concept
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.
Neurogene has an open investigational Gene Therapy Trial for CLN5 and a Natural History Study for CLN5 and CLN7.
Investigational Gene Therapy
Clinical Trial
CLN5
Gene therapy clinical trials are research studies that help determine whether a gene therapy is safe and effective to treat a specific genetic disease
An Investigational Gene Therapy Clinical Trial for CLN5 Batten Disease
Natural History Study
CLN5 and CLN7*
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.
A Natural History Study of Late Infantile Variant CLN5 And CLN7 Disease
*Enrollment for CLN7 patients is full
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