Date: 2017-03-17
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: RegenXBio (USA - MD)
Product: RGX-501
Action mechanism: gene therapy. RGX-501 is being developed as a novel, one-time intravenous treatment for HoFH. RGX-501 is designed to use the NAV AAV8 vector to deliver a functional copy of the LDLR gene to liver cells. This may enable liver cells to make the LDLR protein they need to process elevated levels of LDL cholesterol (LDL-C). The liver is a preferred target for HoFH gene therapy as it is the most important organ for expressing LDLRs, and contributes to greater than 90 percent of the capture and breakdown of LDL-C. RGX-501 has received orphan drug product designation from the U.S. Food and Drug Administration.
Disease: homozygous familial hypercholesterolemia (HoFH)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Country: Canada, USA
Trial details: The Phase I/II first-in-human clinical trial is an open-label, single-center study evaluating the safety and efficacy of RGX-501 in up to 12 patients with homozygous familial hypercholesterolemia in the U.S. and Canada. The primary objective is to assess the safety of a single intravenous administration of RGX-501. The secondary endpoints are the percent of change from baseline of LDL cholesterol at 12 weeks and other lipid outcome measures. This is a dose-escalation study with patients receiving a single dose of 2.5 x 1012 (GC/kg) or 7.5 x 11012 (GC/kg). (NCT02651675)
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