Positive results from phase one gene therapy trial for wet AMD

Posted: Tuesday 22 October 2019

A new study is testing a gene therapy for people with wet age-related macular degeneration (AMD).

The OPTIC study has recently announced positive results from its phase one clinical trial.

Phase one clinical trials test the gene treatment, called ADVM-022, in a small number of people to make sure that it’s safe. This study involved six patients, who were given one dose of the treatment and then monitored for 24 weeks.

The researchers reported that the intravtireal gene therapy had the ability to maintain vision in patients, with a one-time injection and was well-tolerated. These patients previously required frequent anti-VEGF injections to control their wet AMD and to maintain functional vision. ADVM-022 was safe and well tolerated.

Talking about these results, Szilard Kiss, director of clinical research and associate professor at Weill Cornell Medical College, said: "One of the patients in the trial, more interestingly, required 109 injections prior to enrollment. Since receiving the gene therapies, this particular patients has not required any rescue injections with nearly nine months of follow-up.

"Now, these are six patients in the first cohort of the OPTIC trial. More definitely needs to be done, but with a safety profile that we are seeing, which is favorable, and with the efficacy profile that we're seeing, I think within the next 3 to 5 years we can expect gene therapy with anti-VEGF in the office treating our patients who now require frequent injections."

Gene therapy is an exciting area of research, and successful treatments for other eye conditions show that these treatments are possible.

Find out more about gene therapies and how they work.