INVITATION ONLY: GCB-002 gene-therapy study – China

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Status: Enrolling by invitationStudy: GCB-002 in patients with Rett syndrome
Registry number: NCT06739434
Location: Xinhua Hospital, Shanghai, China
Participants: Females aged 2–10 years

What is being studied?
This is a very small, open-label dose-escalation study of a single intrathecal GCB-002 gene-therapy dose. It is designed primarily to assess safety and tolerability, with long-term follow-up planned after treatment.

The registry states that enrolment is by invitation rather than open public recruitment.

Official study record: https://clinicaltrials.gov/study/NCT06739434

Information checked 3 September 2026. This is experimental early-stage research with potential serious and unknown risks. Families should rely on the official investigators and their specialist clinical team.