New One-time Gene Therapy For Wet AMD Could Replace Regular Injections

A new drug called RGX-314 is being developed as a one-time sub-retinal injection for wet AMD. This would be a significant improvement over current anti-VEGF therapies requiring repetitive and frequent intraocular injections. REGENXBIO Inc. has announced that the first human patient has been dosed in a phase I clinical trial of RGX-314. The drug has been shown in animal models to induce a rapid and sustained reduction of VEGF protein in the eyes to provide long-lasting treatment.
RGX-314 differs from current therapeutics in that it includes a gene vector (NAV AAV8) which encodes an antibody fragment designed to neutralize VEGF (vascular endothelial growth factor) activity. This modifies the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss.
Six leading retinal surgery centers across the United States are expected to participate in the Phase I trial of RGX-314. For further details on the trial, enrollment criteria and eligibility, contact [email protected] or visit
Source: REGENXBIO press release