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Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome during ASGCT Annual Meeting

Neurogene Inc. (NGNE) 
Company Research Source: Business Wire
NGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-upNo signs or symptoms of overexpression toxicity, including in one patient with a mild variant predicted to result in residual MeCP2 expressionNeurogene remains on track to provide interim efficacy data from the trial in 4Q:24 NEW YORK--(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 initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome, which showed that NGN-401 was generally well-tolerated by all three patients dosed, with follow-up of approximately nine, six and three months post-dosing. These data were presented during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.“We designed NGN-401 to overcome the limitations of conventional gene therapy Show less Read more
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