OUR PIPELINE
We are building a gene therapy pipeline to help address the high unmet need for rare neurological diseases.
Focusing on the Future of Life-Changing Genetic Medicines
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.
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ABOUT US
At Neurogene, we are driven by a strong sense of purpose to advance genetic medicines.
LATEST NEWS
Neurogene Announces Positive Regulatory Update for NGN-401 Gene Therapy in Rett Syndrome with Plans to Initiate Dosing in Embolden™ Registrational Trial in Q4 2025
Completed discussions with FDA on registrational protocol; 13 sites to allow for rapid enrollment New preclinical data at ESGCT Congress…
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