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Drugs / AAV directed hLDLR gene therapy
last change Nov 2020 re-read 3 minutes ago

AAV directed hLDLR gene therapy

Gene therapy (AAV / viral vector)

Developed for
homozygous familial hypercholesterolemia
Investigated by
National Heart, Lung, and Blood Institute (NHLBI) · REGENXBIO Inc.

Trials 1

today NCT02651675 · Ph 1/2
201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT02651675 Mar 2016 → Nov 2020 homozygous familial hypercholesterolemia REGENXBIO Inc. Terminated No outcome recorded Stop: Business

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 AAV directed hLDLR gene therapy ClinicalTrials.gov intervention name — accepted as the source's own label NCT02651675 ↗
Action Restore “Thus, the functional replacement of the defective LDLR via AAV-based liver-directed gene therapy may be a viable approach to treat this disease and improve response to current...” NCT02651675 ↗
Modality Gene therapy (AAV / viral vector) “AAV (Adeno-associated virus)-based liver-directed gene therapy” NCT02651675 ↗