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 Peer-Reviewed Publication Demonstrating Gene Therapy Candidate NGN-401 with EXACT™ Technology Regulates Transgene Expression in Preclinical Models of Rett Syndrome
Data demonstrate EXACT has potential to overcome limitations of conventional gene therapy for dosage-sensitive disorders Findings published in Science Translational…
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