Drugs / AAVLK03hOTC
last change Jun 2026 re-read 3 minutes ago

AAVLK03hOTC

also known as ssAAV-LK03.hAAT.hcoOTC

Gene therapy (AAV / viral vector)

Developed for
ornithine carbamoyltransferase deficiency
Investigated by
University College, London

Trials 1

202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05092685 Nov 2023 → Jun 2026 overdue ornithine carbamoyltransferase deficiency University College, London Recruiting 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 AAVLK03hOTC ClinicalTrials.gov intervention name — accepted as the source's own label NCT05092685 ↗
Known as ssAAV-LK03.hAAT.hcoOTC “AAVLK03hOTC (also known as ssAAV-LK03.hAAT.hcoOTC)” NCT05092685 ↗
Modality Gene therapy (AAV / viral vector) “new gene therapy (AAVLK03hOTC)” NCT05092685 ↗
Route Intravenous “Peripheral intravenous infusion of AAVLK03hOTC.” NCT05092685 ↗