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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
NEW YORK, April 02, 2025--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 Tran
About this update from Neurogene Inc.
[{"type":"image","alt":"","displaySize":"","headline":null,"caption":"","credit":null,"className":"","disableSlideshowImg":false,"size":{"original":{"width":480,"height":331,"url":"https://media.zenfs.com/en/business-wire.com/79f100278068d1d19edd2a6c4380e9ba"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/UgYKsDIoU3l3XuDxfoun6g--/YXBwaWQ9aGlnaGxhbmRlcjt3PTk2MDtoPTY2MjtjZj13ZWJw/https://media.zenfs.com/en/business-wire.com/79f100278068d1d19edd2a6c4380e9ba","width":480,"height":331}},"lazy":false},{"type":"text","content":"Data demonstrate EXACT has potential to overcome limitations of conventional gene therapy for dosage-sensitive disorders","length":120,"tagName":"p","attribs":{}},{"type":"text","content":"Findings published in Science Translational Medicine","length":52,"tagName":"p","attribs":{}},{"type":"text","content":"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.","length":654,"tagName":"p"},{"type":"text","content":""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 and Chief Executive Officer of Neurogene. "NGN-401 gene therapy for Rett syndrome is our first product candidate incorporating EXACT, which was intentionally developed to deliver therapeutic and tolerable MeCP2 levels. The preclinical data published today and the translation of the platform into the clinic support the proposition that EXACT has the potential to overcome the limitations of conventional gene therapy to address Rett syndrome and other devastating disorders."","length":782,"tagName":"p"},{"type":"text","content":"Rett sy...