New Insight: Restoring Sight With Nanoscope's MCO-010, With Ashish Patel, MD

Welcome back to New Insight with Veeral Sheth, MD!

In this episode of New Insight, host Veeral Sheth, MD, MBA, director of clinical research at University Retina and Macula Associates, speaks with Ashish Patel, MD, senior vice president of sales and marketing at Nanoscope Therapeutics, Inc., to discuss the scientific foundation, clinical evidence, and commercial trajectory of Nanoscope Therapeutics’ emerging optogenetic platform. Sheth and Patel focus on its lead program, MCO-010. Patel begins by outlining his path from academic rhodopsin research to leadership roles in major ophthalmology drug launches across Novartis and Bayer, culminating in his move to early-stage biotech and ultimately to Nanoscope. This background frames the conversation on how a decade of gene-therapy experience informs the development of first-in-class optogenetic treatments for advanced retinal degenerations.

Patel describes Nanoscope as a company uniquely positioned at the forefront of vision-restoration therapeutics, driven by founders who originated the underlying optogenetic technology. He emphasizes the profound unmet need among patients with retinitis pigmentosa (RP), Stargardt disease, and potentially geographic atrophy (GA), where photoreceptor loss leaves patients with severe functional blindness and no restorative treatment options. Unlike mutation-specific gene therapies such as RPE65-targeted Luxturna—applicable to only a small fraction of the RP population—optogenetics offers a gene-agnostic, disease-agnostic strategy. MCO-010 delivers a highly sensitive, broadband opsin to bipolar cells, converting them into surrogate photoreceptors capable of supporting functional vision without requiring external goggles or narrow-spectrum light stimulation.

The discussion highlights extensive clinical data across multiple studies. In an early phase 1/2 open-label RP trial, all treated patients achieved meaningful visual improvements that have been maintained for more than five years with no serious treatment-related safety events. The pivotal RESTORE phase 2b/3 trial demonstrated statistically significant mean gains of approximately three lines of vision (0.3 logMAR) versus sham after a single intravitreal injection—an administration route far simpler than subretinal gene-therapy surgery. 3-year follow-up data presented at AAO confirms durable efficacy. Parallel findings in the STARLIGHT phase 2 Stargardt trial showed an average 12-letter gain in patients treated at the macular disease stage, underscoring the platform’s potential across phenotypes of photoreceptor degeneration.

Patel details Nanoscope’s regulatory progress, including Fast Track and Orphan designations from the US Food and Drug Administration (FDA), a successful pre-BLA meeting, and an active rolling BLA submission for RP. With anticipated priority review, MCO-10 could reach the US market as early as next year. Looking ahead, Nanoscope plans a phase 3 Stargardt program (STARGAZE) and a phase 2 GA trial (Genesis), positioning optogenetics as a transformative platform for restoring functional vision in conditions long considered irreversible.

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