RGX-501

DB17639InvestigationalGene Therapies

RGX-501 is an adeno-associated virus vector 8 (AAV8)-mediated gene therapy that delivers a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells. It is being investigated... It is being investigated for the treatment of homozygous familial hypercholesterolemia.

Code names: RGX-501

What you can answer from here — as of April 02, 2025

1Clinical trialPhase, status and sponsor resolved per trial

1+ReferenceStructured and connected to the statements they support

Which companies are running trials of RGX-501, in which indications and phases, and which of those programs are still active?

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