Nanoscope Therapeutics Reports Five-Year Safety Results from Phase 1/2a Follow-Up Study of MCO-010 Optogenetic Therapy in Retinitis Pigmentosa

DALLAS, Nov. 4, 2025 /PRNewswire/ — Nanoscope Therapeutics Inc. announced today positive long-term safety results from its EXTEND study, a five-year follow-up of participants who received a single intravitreal injection of MCO-010 in an earlier Phase 1/2a trial. 

The EXTEND study followed ten participants with advanced retinitis pigmentosa (RP) who had previously received MCO-010, an optogenetic therapy designed to restore vision using Nanoscope’s proprietary Multi-Characteristic Opsin (MCO) platform.

EXTEND confirmed that a single intravitreal injection of MCO-010 is safe and well-tolerated over five years, with no serious adverse effects or new safety signals, alongside quality-of-life improvements seen over the same five years.

Strong Long-Term Safety Profile

Over five years, the safety profile remained consistent between the Phase 1/2a trial and the long-term follow-up period, with manageable transient inflammation, no discontinuations, and no new safety signals.

“These results demonstrate the excellent long-term safety and tolerability of MCO-010, even five years after a single injection,” said Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope. “The absence of serious safety signals and strong participant retention underscore the potential of MCO-010 as a durable, non-invasive therapy for severe vision loss RP patients.”

Durable Efficacy

During the initial trial, participants who received a higher MCO-010 dose demonstrated statistically significant improvements in visual acuity after one year. Participants continued to report stable or improved vision-related quality of life over the five-year period, particularly in distance activities and vision-specific independence measures. 

The data were presented recently at the 34^th Annual Conference of the Vitreo Retinal Society – India.

About Nanoscope Therapeutics
Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Nanoscope has initiated a rolling BLA submission to the FDA for its lead asset, MCO-010, in the treatment of patients with severe vision loss from retinitis pigmentosa. If approved, MCO-010 has the potential to be the standard of care for RP patients. The company has also shown promising results for MCO-010 to treat Stargardt disease (SD). MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, and macular dystrophies. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start by the end of 2025.

Contact:
Nanoscope Therapeutics
(817) 857-1186
PR@nanostherapeutics.comp 

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