Gene therapy treatment for AMD in clinical trials

Phase 1 clinical trials on a new gene therapy to treat age-related macular degeneration are currently underway in multiple locations around the country.

Early results from the trials are offering promising results and researchers hope gene therapy for wet macular degeneration will be available within the next 3 to 5 years. This new therapy would allow patients to get fewer injections into the eye, which is what the current therapy is for wet macular degeneration. Twelve patients are enrolled in the first two cohorts of the trial and enrollment is continuing for the third and fourth cohorts. Every one of the six patients who have already received the gene therapy has gone at least 6 months without needing anti-VEGF injections. So far, the only adverse reaction noted is mild inflammation that was manageable with steroid eye drops. Controlling inflammation is crucial to the success of the gene therapy. Researchers will finish the phase 1 clinical trial and then begin a bigger, prospective, multi-center controlled clinical trial to continue the gene therapy study.