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

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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. 

Nanoscope Therapeutics Logo

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 34th 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

[email protected] 

Logo – https://mma.prnewswire.com/media/2718911/NSCOPE_nu_Horizontal_2Color_1200px_Logo.jpg 

Cision View original content:https://www.prnewswire.com/in/news-releases/nanoscope-therapeutics-reports-five-year-safety-results-from-phase-12a-follow-up-study-of-mco-010-optogenetic-therapy-in-retinitis-pigmentosa-302603370.html



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Ravindra Soni
Ravindra Sonihttps://businessfortnight.com
Senior journalist with 38 years of experience in print and digital media. Former Executive Editor. Founder and Editor of BusinessFortnight, covering business, economy, and policy news from Madhya Pradesh and across India.
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