Gene therapy may represent future of AMD treatment
Gene therapy represents a new potential avenue for the treatment of retinal diseases, including age-related macular degeneration, according to a presenter at the virtual OSN New York Retina meeting.
“Gene therapy is an exciting new treatment approach in the field of ophthalmology where we can use viral vectors that carry the gene of interest into the host cell,” Aleksandra Rachitskaya, MD, of the Cole Eye Institute, said.
Two therapies undergoing trials for treatment of neovascular age-related macular degeneration, RGX-314 (Regenxbio) delivered subretinally and ADVM-022 (Adverum Biotechnologies) delivered intravitreally, have been mostly well tolerated in early trials, she said.
In the RGX-314 trials, 18 severe adverse events were reported in 11 patients, with one event possibly related to the therapy. Except for one severe case of retinal pigmentary changes, all other common ocular adverse events were mostly mild.
In the ADVM-022 trials, all ocular adverse events were deemed mild to moderate, with the biggest concern being inflammation, which has been managed with steroid eyedrops, Rachitskaya said.
So far, early data in these ongoing trials show potential for reduced need for anti-VEGF treatments after gene therapy, Rachitskaya said. Annualized injection rates, numbers of rescue injections, treatment effect in previously treated vs. treatment naive patients, and effect on visual acuity are among efficacy measures to be considered.
“Safety is paramount, and we have seen some concerns of inflammation, IOP elevation and pigmentary changes,” Rachitskaya said. “We have to wait for the final results to see if this is significant.”
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Rachitskaya A. Gene therapy for AMD. Presented at: OSN New York and OSN New York Retina; Oct. 17-18, 2020 (virtual meeting).