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

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Neurogene Inc. has made significant progress in its NGN-401 gene therapy for Rett syndrome, with multiple participants dosed in the Embolden trial. Dosing is expected to conclude by Q2 2026, and interim safety and efficacy data will be presented in mid-2026. The company is also preparing for early commercial activities and has reported positive interim results from the Phase 1⁄2 trial. Neurogene will present at the J.P. Morgan Healthcare Conference on January 14.
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