New research paper highlights the unique structure and function of Nanoscope’s synthetic opsin, supporting its potential in vision restoration
DALLAS, July 22, 2025 /PRNewswire/ — Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene therapies for patients suffering vision loss from retinal degenerative diseases, today announced the publication of a paper “Topology and Mechanism of Broadband and Fast Multi-Characteristic Opsin (MCO) for Neuromodulation,” in Advanced Therapeutics, a peer-reviewed journal in Wiley’s prestigious Advanced portfolio, known for showcasing high impact biomedical research. The paper details the biophysical characteristics of Nanoscope’s proprietary therapeutic approach, MCO, including its unique structure and function, which utilizes a combination of predictive protein modeling, molecular assays, and electrophysiology.
The paper highlights Nanoscope’s groundbreaking research that supports the impact observed in patients treated with the company’s lead asset therapy, MCO-010. The findings lay the groundwork to further advance this technology to help individuals affected by a range of vision loss due to retinal degenerative diseases. Functional studies using electrical recordings demonstrated that MCO is activated by ambient light, exhibits fast on-off kinetics with high photosensitivity, and maintains a broad dynamic range across the visible spectrum, enabling robust, best-in-class light-activation of MCO-expressing cells.
“The highly efficient mechanism of action of our ambient light-activatable MCO stems from its unique structure, which allows broadband light sensitivity combined with fast channel kinetics,” said Dr. Samarendra Mohanty, President & Chief Scientific Officer at Nanoscope and lead author of the paper. “Based on this research and multiple clinical studies, a rolling submission was recently initiated for a Biologics License Application to the FDA for MCO-010, with the potential to be the first gene-agnostic therapy to treat retinitis pigmentosa.”
The unique topological and functional activation properties of the fused MCO protein represent novel findings that link both in vitro and in vivo efficacy of this opsin, critical for neuromodulation aimed at restoring vision in patients with retinal degenerative diseases.
Dr. Ione Fine, Professor, Department of Psychology at the University of Washington, Seattle, commented, “The paper provides a comprehensive comparison of the performance of MCO with other opsins in development for treating retinal degenerative diseases. As such, it provides critical insights to support computational modeling for predicting sight recovery in patients treated with MCO and other opsins.”
Click here to access the published paper.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapy for the millions of patients blinded by retinal degenerative diseases. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office intravitreal injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 therapy in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025. MCO-010 has received FDA Fast Track designations and orphan drug designations for both RP and SD. Preclinical programs include Leber congenital amaurosis (LCA), in IND-enabling studies, as well as an IND-ready asset for geographic atrophy (GA).
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SOURCE Nanoscope Therapeutics
