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A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)

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Trial Profile

A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 25 May 2026

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At a glance

  • Drugs NGN 401 (Primary)
  • Indications Rett syndrome
  • Focus First in man; Registrational; Therapeutic Use
  • Acronyms Embolden; Embolden (TM)
  • Sponsors Neurogene Inc

Most Recent Events

  • 12 May 2026 According to a Neurogene media release, on track to complete dosing in the second quarter of 2026 ~90% of participants have been dosed, the company will present updated interim safety and efficacy data on the pediatric and adolescent/adult cohorts from the Phase 1/2 trial, including at least 12 months of follow-up for all 10 participants, in mid-2026.
  • 26 Feb 2026 According to a Neurogene media release, completion of dosing expected in the second quarter of 2026.
  • 26 Feb 2026 According to a Neurogene media release, based on interim NGN-401 Phase 1/2 data the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NGN-401, an investigational gene therapy in late-stage clinical development as a potential best-in-class treatment for Rett syndrome.

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