RGX-501

A single treatment with the potential
for long-lasting results

diver

RGX-501 is our product candidate for the treatment of homozygous familial hypercholesterolemia (HoFH).

RGX-501 is designed to use the AAV8 vector to deliver the human low-density lipoprotein receptor (LDLR) gene to liver cells.

HoFH is a monogenic disorder caused by abnormalities in the function or expression of the LDLR gene. HoFH patients have very low levels or are completely deficient of LDLR, resulting in very high total blood cholesterol levels. This leads to premature and aggressive plaque buildup, life threatening coronary artery disease (CAD) and aortic valve disease.

The current standard of care in HoFH focuses on early initiation of aggressive treatment due to severe clinical effects of elevated LDL-C. Available therapies do not provide a cure and their use is limited due to tolerability and drug availability.

We believe that the liver is the preferred target organ for gene therapy of HoFH since LDLRs produced in the liver contribute to greater than 90 percent of the capture and breakdown of LDL, making the liver by far the most important LDLR producing organ.

Patients with HoFH are currently being enrolled in a Phase I/II clinical trial of intravenously administered RGX-501. RGX-501 has received orphan drug product designation from the FDA.