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Neurogene Announces Peer-Reviewed Publication Demonstrating Gene Therapy Candidate NGN-401 with EXACT™ Technology Regulates Transgene Expression in Preclinical Models of Rett Syndrome [Yah...

Neurogene Inc. (NGNE) 
Company Research Source: Yahoo! Finance
Findings published in Science Translational Medicine NEW YORK, April 02, 2025 BUSINESS WIRE )--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced a peer-reviewed publication on its EXACT™ transgene regulation technology. The preclinical studies demonstrate EXACT's ability to regulate gene therapy expression to a tolerable and therapeutic level, including in preclinical models of Rett syndrome. These data, published in Science Translational Medicine , supported the initiation of Neurogene's ongoing Phase 1/2 clinical trial with NGN-401 gene therapy for Rett syndrome. "Neurogene was founded to extend the promise of gene therapy to rare neurological disorders, and EXACT was critical to enabling gene therapy treatment for Rett syndrome, a disorder with a narrow therapeutic window that requires transgene regulation," said Rachel McMinn, Ph.D., Founder Show less Read more
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