OUR PIPELINE
We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
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.
Learn More >>We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
At Neurogene, we are driven by a strong sense of purpose to advance genetic medicines.
Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates
Announced design of Embolden™ registrational clinical trial of NGN-401 gene therapy for Rett syndrome; trial initiation activities underway Completed dosing…
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