ACTIVE: NGN-401 EXACT regulated gene-therapy study – Australia, UK and USA

Status: Active, not recruitingStudy: EXACT study of NGN-401
Registry number: NCT05898620
Countries: Australia, United Kingdom and United States
Participants: Females aged 3 years and older

What is being studied?
NGN-401 is an investigational AAV gene therapy designed to deliver MECP2 using Neurogene’s regulation technology. The study is evaluating safety, tolerability and preliminary clinical effects after a one-time administration.

The study remains active but is not currently recruiting according to the registry.

Official study record: https://clinicaltrials.gov/study/NCT05898620

Information checked 3 September 2026. Gene therapy is experimental and may involve serious or unknown risks. Families should confirm developments through the official record and their Rett specialist.