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 Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome
Written agreement from FDA for single-arm, open-label, baseline-controlled registrational trial evaluating composite primary endpoint of improvement in CGI-I and gain…
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