Nanoscope Therapeutics presented three-year follow-up data from its REMAIN study of RESTORE Phase 2b trial, focusing on MCO-010 (sonpiretigene isteparvovec), at the 2025 Euretina Congress in Paris and the Retina Society Annual Scientific Meeting in Chicago. The company said that the data demonstrated positive efficacy and safety outcomes over 152 weeks for its lead optogenetic therapy targeting retinitis pigmentosa.
MCO-010, delivered via a single in-office intravitreal injection, is the first therapy in a randomized, controlled trial to restore vision in patients with severe vision loss due to retinitis pigmentosa, according to the company. The company noted that the durability of effect and tolerability shown in the long-term follow-up could reshape the standard of care for this condition.
The presentations lay the groundwork for the company’s rolling Biologics License Application submission to the U.S. Food and Drug Administration, which has already been initiated. Additional clinical development plans include a Phase 3 registrational trial in Stargardt disease in 2025, and a planned Phase 2 program for geographic atrophy by year-end; MCO-010 holds both FDA Fast Track and Orphan Drug designations, as well as EMA Orphan designation covering multiple retinal dystrophies.
