ReGenX Bio has received U.S. FDA approval to begin a phase 1 multi-center trial for their NAV vector based candidate RGX-314 for treatment of wet AMD.
RGX-314 uses a NAV AAV8 vector to deliver a gene encoding a monoclonal antibody fragment to retinal cells that, when expressed, neutralizes VEGF activity. ReGenX says its NAV vector platform improves upon previous AAV vector therapies by increasing the level and duration of gene expression in transduced cells.
“The goal of the RGX-314 program is to develop a single-dose treatment for wet AMD that prevents future disease recurrence while reducing or eliminating the need for regular injections that are the current standard of care in wet AMD,” said Kenneth T. Mills, ReGenX Bio president and CEO. “We are on track to meet our next program objectives for RGX-314, beginning with trial enrollment by mid-2017 and an interim trial update by the end of the year.”
In preclinical animal models of macular degeneration, a single subretinal dose of RGX-314 resulted in significant and dose-dependent reduction of blood vessel growth and prevention of disease progression.
“In animal studies, treatment with RGX-314 gene therapy led to rapid and sustained anti-VEGF protein detected in the eyes of treated animals,” said Albert Maguire, MD, one of the collaborating researchers. “Preclinical studies have shown anti-VEGF mRNA and protein distributed widely throughout the retina. This high protein expression observed using RGX-314’s NAV AAV8 vector may make this approach suitable for an ocular therapeutic in wet AMD.”
Six leading retinal surgery centers across the United States are expected to participate in the Phase I trial of RGX-314, including: James M. Wilson, MD, PhD, Jean Bennett, MD, PhD, and Albert Maguire, MD, from the University of Pennsylvania’s Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics, and Peter Campochiaro, MD, at the Johns Hopkins Wilmer Eye Institute.