While treatments such as Lucentis®, Avastin®, and Eylea® have been saving and restoring vision for people with wet age-related macular degeneration (AMD) over the last several years, they have a significant drawback: The therapies require regular injections into the eye—in some cases, monthly—for the life of the patient.
But there was good news reported during ARVO’s annual meeting last week, about an early-stage clinical trial of a wet AMD gene therapy known as RetinoStat® that requires far fewer injections. While researchers are still learning about the long-term efficacy of the treatment, they believe one injection will last for several years, perhaps the lifetime of the patient.
Oxford BioMedica, the developer of RetinoStat, reported that the treatment was safe and expressed the desired therapeutic proteins during the 48-week, 21-patient, Phase I study. Most exciting, patients had stable visual acuity. Also, the vision-robbing, vascular leakage that’s the hallmark of wet AMD was reduced. The study results were selected as an ARVO meeting “Hot Topic.”
Phase I studies are primarily focused on safety, so there is a long way to go before Oxford BioMedica can conclude that the treatment works and seek regulatory approval. Many more patients need to be evaluated over longer time periods. But these results are encouraging.
With earlier, preclinical funding from the Foundation Fighting Blindness, Oxford BioMedica initiated commercial development of three retinal-disease gene therapies—RetinoStat as well as StarGen™, for Stargardt disease, and UshStat®, for Usher syndrome type 1B. In 2014, the international pharmaceutical company Sanofi acquired development and commercialization rights to StarGen and UshStat. These early-stage clinical trials are ongoing, and we’ll report results when the company makes them available.
Pictured, above: A retina affected by the hemorrhaging associated with wet AMD. Photo courtesy of the National Eye Institute.