Drugs / AAV9.hMCOLN1co
last change Jan 2026 re-read 3 minutes ago

AAV9.hMCOLN1co

Gene therapy (AAV / viral vector)

Developed for
mucolipidosis type IV
Investigated by
The Children's Hospital of Zhejiang University School of Medicine

Trials 1

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT07398872 Jan 2026 → Jan 2027 expected mucolipidosis type IV The Children's Hospital of Zhejiang University School of Medicine Enrolling by invitation No outcome recorded

Evidence & citations 3 cited values

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

FieldValueCited text
Known as AAV9.hMCOLN1co ClinicalTrials.gov intervention name — accepted as the source's own label NCT07398872 ↗
Modality Gene therapy (AAV / viral vector) “AAV9. hMCOLN1co, a gene therapy product” NCT07398872 ↗
Route Intrathecal “A single intrathecal infusion” NCT07398872 ↗