Coming off the heels of the first ocular gene therapy approved in the United States, Szilard Kiss, MD, pointed out that the gene therapy era for retinal disease has arrived, “and hopefully it will arrive for age-related macular degeneration (AMD).”
When clinicians and researchers think about gene therapy, it is not just one product, but four different factors, said Dr. Kiss, assistant professor of ophthalmology; director of clinical research, Weill Cornell Medical College, New York. Considerations include the virus used (or if a virus will be used for delivery), and what is actually being delivered.
“We need to consider if we’re delivering anti-vascular endothelial growth factor (anti-VEGF) aflibercept or anti-VEGF ranibizumab or a gene therapy to treat a rare disorder?” Dr. Kiss said. “How are you delivering that in terms of formulation? Where in the retina–either intravitreal or subretinal space–are you delivering your gene therapy vector?”
Monogenetic disorders (such as Leber’s congenital amaurosis) readily lend themselves to gene therapy, Dr. Kiss believes.
“It’s amazing that we can cure blindness in young children,” Dr. Kiss said. “But what would be even more amazing is if we could somehow alleviate the 6 million injections we’re doing every year for things like diabetes and macular degeneration.”
Gene therapy itself is “not a new concept,” with 3 phase I clinical trials for AMD having been completed. Those studies did not move to phase II “for a variety of reasons,” Dr. Kiss cited. “But what they did show is that subretinal delivery in wet AMD is safe and feasible.”