Gene therapy holds incredible promise to revolutionize medical treatment. At Neurogene we believe that innovation, robust scientific execution and a strong sense of purpose are required to turn this promise into reality. Our focus is to develop life-changing genetic medicines for people and their families impacted by devastating neurological diseases.

A passion for serving patients and their families

We listen to patients and families and incorporate their perspectives into our work, striving to support them throughout our research and development process and beyond.

Gene therapies for rare neurological disorders

We are advancing a gene therapy pipeline to help address the high unmet need for rare neurological diseases, with a focus on Rett syndrome.

A differentiated platform technology

We leverage our proprietary transgene regulation technology to open up new possibilities for rare neurological diseases.

Girl with Rett syndrome

Pursuing a Brighter Future, Together

We own our responsibility to provide transparent updates on our progress on an ongoing basis to those in the patient communities we serve. This partnership is critical to advance a potential one-time treatment for a rare neurodevelopmental disorder like Rett syndrome.

Patients and families are at the core of our work.

We recognize and honor that it is their contributions that make scientific advances possible. We strive to support them throughout their participation in clinical research.

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