Neurogene Advances NGN-401 Gene Therapy for Rett Syndrome with Embolden Trial Dosing and Upcoming 2026 Milestones

Reuters01-12

Neurogene Advances NGN-401 Gene Therapy for Rett Syndrome with Embolden Trial Dosing and Upcoming 2026 Milestones

Neurogene Inc. announced recent progress in its NGN-401 gene therapy program for Rett syndrome. Multiple participants have been dosed in the Embolden registrational trial, with dosing expected to be completed in the second quarter of 2026. The company plans to present interim safety and efficacy data from its Phase 1/2 trial, including at least 12 months of follow-up for all participants in the pediatric and adolescent/adult cohorts, in mid-2026. Early commercial-readiness activities are underway, and Neurogene has reported positive interim data from the Phase 1/2 trial, demonstrating durable multidomain improvements across a spectrum of disease severity. The company is scheduled to present at the J.P. Morgan Healthcare Conference on January 14.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20260112556591) on January 12, 2026, and is solely responsible for the information contained therein.

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Multiple participants have been dosed in the Embolden registrational trial, with dosing expected to be completed in the second quarter of 2026. The company plans to present interim safety and efficacy data from its Phase 1/2 trial, including at least 12 months of follow-up for all participants in the pediatric and adolescent/adult cohorts, in mid-2026. Early commercial-readiness activities are underway, and Neurogene has reported positive interim data from the Phase 1/2 trial, demonstrating durable multidomain improvements across a spectrum of disease severity. The company is scheduled to present at the J.P. Morgan Healthcare Conference on January 14.\n      \n\n\nDisclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. 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