Neurogene Doses First Participant in Embolden™ Registrational Trial of NGN-401 for Rett Syndrome

Neurogene announced today that the first participant has been dosed in their registrational clinical trial evaluating NGN-401, a gene therapy for Rett syndrome.

Key details:

• Single-arm, open-label, study in 20 females aged three years and older.
• Enrollment expected to complete within three to six months at 13 US clinical sites
• Trial intended to support a Biologics License Application (BLA) — a formal request to the FDA to approve a biological product, such as a gene therapy, for clinical use.
• Community letter to come shortly