US-based clinical-stage biotechnology firm Regenxbio has commenced dosing in a Phase I/II clinical trial of its gene therapy RGX-501 to treat homozygous familial hypercholesterolemia (HoFH).
RGX-501 is an investigational therapy that utilises the NAV AAV8 vector to deliver a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells.
It is expected that the gene may enable liver cells to make the LDLR protein to process LDL cholesterol.
The open-label, single-centre, dose-escalation Phase I/II trial will evaluate the safety and efficacy of RGX-501 in approximately 12 patients within the US and Canada.
Regenxbio founder and chief executive officer Kenneth Mills said: "Regenxbio is pleased to announce that we have advanced into the clinic with the dosing of the first patient in one of our lead development programmes.
"This significant milestone is a tribute to the hard work of teams at Penn and Regenxbio to bring this potentially lifechanging therapy to patients with HoFH.
“We look forward to continuing enrolment and expect to report interim trial results by the end of 2017.”
The trial's primary objective is to determine the safety of a single intravenous administration of RGX-501, while the secondary endpoints include the percentage of change from baseline of LDL cholesterol at 12 weeks, as well as other lipid outcome measures.
The US Food and Drug Administration (FDA) has also granted orphan drug product designation to RGX-501.
The firm's adeno-associated virus (AAV) gene delivery platform, known as NAV Technology, includes more than 100 new AAV vectors such as AAV7, AAV8, AAV9 and AAVrh10.