Drugs / GCB-002
last change Nov 2024 re-read 3 minutes ago

GCB-002

Gene therapy (AAV / viral vector) targets MECP2

Developed for
Rett syndrome
Investigated by
Genecombio Ltd. · Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Trials 1

PhaseRegistry idDatesIndicationSponsorStatusOutcome
— NCT06739434 Nov 2024 → Dec 2026 expected Rett syndrome Genecombio Ltd. Enrolling by invitation No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as GCB-002 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06739434 ↗
Modality Gene therapy (AAV / viral vector) “self-complementary AAV9 carrying a full length human MECP2 transgenetic product” NCT06739434 ↗
Route Intrathecal “single intrathecal injection” NCT06739434 ↗
Target MECP2 “full length human MECP2 transgenetic product” NCT06739434 ↗