Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome
Two pediatric patients with Rett syndrome dosed in the United States with NGN-401, Neurogene’s lead gene therapy product candidate leveraging its proprietary EXACT gene regulation technology
NGN-401 has been well-tolerated to date with no treatment-emergent or procedure-related serious adverse events (SAEs) or transgene-related overexpression toxicity
Third patient dosing anticipated in 1Q24, and interim efficacy data from multiple patients in the first cohort remains on track for 4Q24
NGN-401 design and delivery approach optimized to maximize efficacy and safety profile
https://www.neurogene.com/press-rel...female-pediatric-patients-with-rett-syndrome/
Media News, page-206
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