Nanoscope Therapeutics presented groundbreaking research on its lead optogenetic therapy MCO-010 at the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT 2025) in New Orleans, showcasing data across multiple retinal degenerative diseases and advancing toward commercialization.
Clinical Progress Across Multiple Indications
The biotech company highlighted safety and efficacy results for MCO-010 across several retinal conditions, including clinical trial data in Stargardt macular degeneration and non-human primate studies in geographic atrophy. According to Dr. Samarendra Mohanty, co-founder and President of Nanoscope, the positive therapeutic benefits observed in Stargardt patients were similar to the durable vision gains previously demonstrated in patients with retinitis pigmentosa (RP).
MCO-010 represents the most advanced program in Nanoscope's MCO (multi-characteristic opsin) platform and stands as the first and only mutation-agnostic therapy shown to restore vision in advanced RP patients through a one-time, standard intravitreal injection.
Comprehensive Research Presentations
The company delivered multiple presentations spanning three days of the conference, including podium presentations on safety and efficacy in non-human primate models of geographic atrophy, laser-based non-viral delivery methods, and potency assay development. Poster presentations covered human retinal cell delivery in Stargardt disease patients, forced degradation studies of the gene therapy drug product, and regulatory pathway considerations.
Dr. Mohanty emphasized the company's focus on presenting "robust assays for ensuring the potency of MCO-010" alongside the clinical benefits data, indicating a comprehensive approach to therapy development and quality control.
Regulatory Milestones and Commercial Timeline
Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), Nanoscope announced plans to initiate a BLA submission for MCO-010 to treat RP in 2025. The company has also completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025.
MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt disease, providing regulatory advantages for development and potential approval.
Expanding Pipeline and Technology Platform
Beyond MCO-010, Nanoscope's preclinical programs include an IND-ready non-viral laser-delivered MCO-020 asset for geographic atrophy and an AAV asset for Leber Congenital Amaurosis currently in IND-enabling studies. The company positions itself as developing mutation-agnostic, sight-restoring optogenetic therapies for millions of patients blinded by retinal degenerative diseases for which no treatment currently exists.
