Despite having a checkered past, gene therapy seemed like a promising option for many patients with this disease. There were more volunteers than could be accommodated in this trial, Cideciyan noted.
But the treatment isn't out of the woods yet. The research team will continue the study for a full three years, as is required by the U.S. Food and Drug Administration (FDA) for a phase I clinical trial. And the participants will be monitored for 15 years for health impacts, as is required by the FDA for any gene-therapy trials. Following the end of the first trial, there will be two more—to test the treatment's efficacy—before it can be approved for commercial use on the market.
Cideciyan admits that the encouraging results are only for a rare condition, but he hopes that with continued research, it will "have huge potential for much more common diseases."