Transgene Regulation
CNS + Ocular Delivery
IND* Enabling
Phase I/2
Expected Milestones
Preliminary Data
Additional Data 2H:25
Preliminary Data

*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.

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Clinical Trials

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.

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Gene Therapy Study for Children With CLN5 Batten Disease

Gene therapy clinical trials are research studies that help determine whether a gene therapy is safe and effective to treat a specific genetic disease

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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.

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Expanded Access Policy

Neurogene is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. To accomplish this, we collaborate with many researchers, the patient community, patient advocacy organizations, and physicians, in addition to working closely with global regulatory agencies. With the development of medicines that will be safe, effective, and satisfy unmet medical need, comes the promise that these medicines will be broadly accessible to appropriate patients in the future.

Our Policy

Currently, Neurogene does not offer an expanded access program.

Neurogene carefully considered our policy on providing access to investigational gene therapies outside of a clinical trial. We took into account multiple considerations, including patient safety, potential risks and benefits, the impact to the broader patient population, resourcing, and other ethical considerations. At this time in development, availability and access to Neurogene investigational therapies is possible only through participation in clinical trials. Clinical trials are carefully regulated, monitored, and conducted in specific patient populations to assess safety, and serve a critical role in understanding the risks and potential benefits of investigational therapeutic options, such as gene therapy. For these reasons, we believe that completing the necessary clinical trials and obtaining the data needed for review and approval by regulatory agencies is the fastest and most scientifically sound way toward making Neurogene’s gene therapies available to the broader patient population.

If you are a patient or family member and have questions about this policy, please contact If you are interested in learning more about our current investigational gene therapy programs, visit here or our Patients and Families page.

If you are a healthcare provider and have questions about this policy, please contact