An investigational gene therapy for geographic atrophy in advanced age-related macular degeneration (AMD) has continued to show good toleration results and no serious side effects, according to data presented at the Retina Society’s 54th Annual Scientific Meeting, which was held September 29 to October 2 in Chicago. Szilárd Kiss, MD, associate professor of ophthalmology and director of clinical research at Weill Cornell Medical College in New York, reported interim data from the ongoing phase 1/2 FOCUS trial of GT005, a gene therapy delivered by subretinal injection that was developed by Gyroscope Therapeutics, a UK-based biotech company.

Of 28 patients enrolled in FOCUS, none showed dose-related trends in the frequency or type of adverse events, as well as no serious side effects related to GT005. In a subset of 13 patients who had received the gene therapy at least 29 weeks before the analysis, 11 showed increases in vitreous levels of complement factor I (CFI), a protein shown to downregulate complement system activity. These patients also showed decreased levels of the complement breakdown proteins Ba and C3, with all complement-related activity limited to the eye.

“These data build on the positive interim results we shared earlier this year, reinforcing GT005’s potential to restore balance to an overactive complement system and continuing to show an encouraging safety profile,” said Nadia Waheed, MD, MPH, Gyroscope’s chief medical officer. “It’s an exciting time in complement research, and we believe a one-time gene therapy has the potential to be a long-term solution for people with geographic atrophy.”