Nanoscope Announces 3-Year Vision Improvements from REMAIN Study of MCO-010 in RP
Nanoscope Therapeutics announced 3-year follow-up data from the REMAIN study, the long-term extension of its pivotal phase 2b/3 RESTORE trial evaluating MCO-010, the company’s proprietary optogenetic therapy for retinitis pigmentosa (RP).
The REMAIN study demonstrated that a single intravitreal injection of MCO-010 produced clinically meaningful and durable vision improvements in patients with RP, sustained through 152 weeks. Participants maintained an average best corrected visual acuity (BCVA) gain of approximately 0.3 LogMAR, equivalent to an improvement of 3 lines (15 letters) on a standard ETDRS vision chart. Compared with sham-treated controls, the BCVA-Area Under the Curve (AUC) profiles showed a fivefold greater vision gain across the combined RESTORE and REMAIN studies.
Importantly, MCO-010 continued to exhibit a favorable safety and tolerability profile, with no serious ocular adverse events reported. Only one mild case of inflammation required short-term topical steroid treatment, and 14 of 15 treated patients required no ongoing inflammation management at Week 152.
“The sustained visual gains demonstrated over 3 years are truly remarkable for this population,” said Allen C. Ho, MD, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor for Nanoscope. “These patients typically lose about 1.5 lines of vision every 5 years and often reach legal blindness as early as age 20. The ability to not just slow loss but restore visual function for several years represents a significant therapeutic advance.”
“These long-term results underscore the lasting impact of MCO-010 in patients who currently have no treatment options,” said Sulagna Bhattacharya, CEO of Nanoscope Therapeutics. “As we advance our rolling Biologics License Application (BLA) with the FDA for MCO-010 in RP, we are actively preparing for a potential commercial launch of what could become the first approved optogenetic therapy for retinal disease. The treatment durability observed in REMAIN also highlights the potential of our platform as a one-time therapy for a broad range of retinal degenerative conditions.”
Presentations at Ophthalmology Conferences
The REMAIN study findings were recently presented at several ophthalmology meetings, including:
American Academy of Ophthalmology (AAO) 2025 Annual Meeting – Allen C. Ho, MD, Wills Eye Hospital, Philadelphia, PA
Retina Society 58th Annual Scientific Meeting – Christine Kay, MD, Vitreo Retinal Associates, Gainesville, FL
25th EURETINA Congress – Jordi Mones, MD, PhD, Institut de la Màcula, Barcelona, Spain
Nanoscope’s Multi-Characteristic Opsin (MCO) platform is a one-time, intravitreal, disease-agnostic therapy designed to restore vision in patients with advanced retinal degenerations, including retinitis pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). The technology works by activating dense bipolar retinal cells to become light-sensitive, leveraging the retina’s remaining visual circuitry after photoreceptor loss. The approach avoids the need for genetic testing, invasive surgery, or repeat dosing, allowing integration into standard retina clinic workflows.
MCO-010 has received Fast Track and Orphan Drug designations from the FDA; and the European Medicines Agency (EMA) has granted five Orphan designations for MCO-010 across multiple retinal degenerative conditions.