August 11, 2020

Gene therapy treatment for wet AMD shows positive results


Gene therapy treatment for wet AMD shows positive results

(Updated 12/11/2020)

REGENXBIO, Inc. has reported positive one year data from patients in Cohorts 4 and 5 of the Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). As reported here in June 2017, RGX-314 is being developed as a one-time sub-retinal injection for wet AMD. It would be a significant improvement over current anti-VEGF therapies requiring repetitive and frequent intraocular injections. 

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.

“Based on the overall results to date from the Phase I/IIa trial, I believe that RGX-314 has the potential to profoundly impact all aspects of clinical management for patients with wet AMD,” said Robert Avery, M.D., Founder of California Retina Consultants and Research Foundation and investigator surgeon in the trial. “Wet AMD affects a large number of adults, and often results in loss of vision over time due to non-compliance with the current standard of care of frequent anti-VEGF injections. I am encouraged that RGX-314 has the potential to become a one-time gene therapy treatment option for a broad range of patients.”

REGENXBIO has announced dosing of the first patient in the Phase II ALTITUDETM trial of RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery. REGENXBIO expects to report initial data in 2021

For further details on the trial, enrollment criteria and eligibility, visit

SOURCE:  Press release

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