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 Announces Peer-Reviewed Publication Demonstrating Gene Therapy Candidate NGN-401 with EXACTâ„¢ 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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