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