Trial Profile

A registrational study of NGN-401 Gene Therapy for Treatment of Rett Syndrome

Status: Planning

Phase of Trial: Phase III

Latest Information Update: 01 Apr 2025

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At a glance

Drugs NGN 401 (Primary)
Indications Rett syndrome
Focus Registrational; Therapeutic Use
Sponsors Neurogene Inc

Most Recent Events

24 Mar 2025 According to a Neurogene media release, company remains on track to provide a regulatory update on registrational trial plans in the first half of 2025 and additional participants expected to be dosed during the first half of the year
11 Nov 2024 According to a Neurogene media release, company anticipate providing an update on the trial design in the first half of 2025.
21 Mar 2024 New trial record

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