Conventional Gene Therapy
Product
Candidate
Indication
Discovery
In Vivo POC**
IND Enabling
Phase I/II
Batten Disease
NGN-101
NGN-102
Undisclosed
NGN-103
Leukodystrophy
NGN-201
Neurodevelopmental
NGN-402
Undisclosed

*EXACT = Expression Attenuation via Construct Tuning
**POC = Proof of Concept

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.

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Clinical Trials

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

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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

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