Neurogene Doses First Participant in Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome

Reuters11-06

Neurogene Doses First Participant in Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome

Neurogene Inc. has announced the dosing of the first participant in the Embolden registrational clinical trial of NGN-401, a gene therapy candidate for the treatment of Rett syndrome. Embolden is a single-arm, open-label, baseline-controlled trial evaluating a 1E15 vector genome dose of NGN-401 in 20 females aged three years and older. Enrollment for the trial is underway in the U.S., with completion expected within three to six months. Interim data from the Phase 1/2 trial of NGN-401 will be presented by company management at the Stifel 2025 Healthcare Conference on November 12, 2025.

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: 20251106135659) on November 06, 2025, and is solely responsible for the information contained therein.

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