Nanoscope Begins Rolling BLA for MCO-010 in RP

Nanoscope Therapeutics has announced the initiation of a rolling submission of a biologics license application (BLA) to the US Food and Drug Administration (FDA) for its lead investigational therapy, MCO-010, targeting severe vision loss due to retinitis pigmentosa (RP).

A First Gene-Agnostic Gene Therapy for Retinal Disease

This milestone represents the first BLA submission for a gene-agnostic gene therapy in retinal disease, according to the company. Being gene-agnostic means MCO-010 is designed to address the broad genetic diversity of RP, a condition associated with more than 100 genes and over 1,000 different mutations, independent of the specific underlying genetic cause.

The FDA has granted Nanoscope rolling review of the BLA, with initial modules already submitted. Full submission is anticipated by early 2026, and the application qualifies for priority review under its fast-track designation.

If approved, MCO-010 would become the first gene-agnostic therapy available to restore vision in legally blind RP patients.

Overview of MCO-010’s Delivery and Mode of Action

MCO-010 is based on Nanoscope’s proprietary multi-characteristic opsin (MCO) platform and is administered via a single, in-office intravitreal injection. After delivery, MCO-010 activates surviving bipolar retinal cells to become light-sensitive, leveraging the remaining visual pathways even after photoreceptor loss.

Unlike many gene therapies, MCO-010 is designed to eliminate the need for genetic testing, surgical procedures, or repeat dosing. This approach could make it broadly applicable across the RP patient population and integrate seamlessly into existing retina practice workflows.

Leadership Insights on Progress Toward Approval

“For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight,” said Sulagna Bhattacharya, CEO and Co-Founder of Nanoscope. “We are deeply thankful to the FDA for their guidance as we remain steadfast in our mission to restore vision and bring light back into the lives of those living in darkness.”

Samarendra Mohanty, PhD, President, Chief Scientific Officer, and Co-Founder of Nanoscope, added:

“We’ve been working on the MCO platform for more than a decade. We’ve seen this investigational therapy surpass our expectations in the lab and in patients in clinical trials, and we believe we’re now one step closer to potentially bringing this pioneering therapy to all RP patients.”

RP’s Impact and MCO-010 Clinical Results

RP is one of the leading causes of blindness among the working-age population in the US, affecting more than 100,000 people, with over 25,000 considered legally blind. On average, RP patients lose one line of vision on an eye chart every three years, with most reaching legal blindness (worse than 20/200 vision) by age 60.

In the pivotal RESTORE phase 2b trial, MCO-010 met its best corrected visual acuity (BCVA) primary endpoints in both dose groups at 52 weeks compared to sham control. Patients demonstrated vision gains from baseline of greater than 0.3 LogMAR, equivalent to an improvement of three or more lines on an eye chart. Long-term follow-up has shown these gains continue through three years, with no serious adverse events reported in treated eyes. Further multi-year follow-up is planned to continue evaluating long-term efficacy and safety.