Nanoscope Initiates Rolling Submission of BLA to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat RP

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

This marks a significant regulatory milestone as the first BLA submitted for a gene-agnostic gene therapy for retinal disease. According to Nanoscope, being gene-agnostic means that MCO-010 is designed to address the broad genetic diversity of RP—a condition linked to over 100 known genes and more than 1,000 different mutations—regardless of the underlying mutation.

The FDA has granted Nanoscope rolling review of its BLA, with the first modules already submitted and completion of the full BLA submission anticipated in early 2026. The application is eligible for priority review under the program's fast-track designation.

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

"For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight," Sulagna Bhattacharya, CEO and Co-Founder of Nanoscope, said in a company news release. "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."

MCO-010, Nanoscope's proprietary multi-characteristic opsin (MCO), is delivered via a one-time, in-office intravitreal injection. After injection, MCO-010 activates highly dense bipolar retinal cells to become light sensitive, utilizing remaining visual circuitry following photoreceptor death. MCO-010 is designed to offer a distinct approach in that it does not require genetic testing, surgical intervention, or repeat dosing, making it applicable to a broad population of RP patients, while also fitting into existing retina office workflows.

"We've been working on the MCO platform for more than a decade," said Samarendra Mohanty, PhD, President, Chief Scientific Officer, and Co-Founder of Nanoscope. "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."

According to Nanoscope, RP is one of the leading causes of blindness in the working-age population in the US, affecting more than 100,000 people and leaving over 25,000 legally blind. Vision loss due to RP is highly dependent on the underlying mutation present, and, on average, RP patients lose 1 line of vision on an eye chart approximately every 3 years, and a majority are legally blind (worse than 20/200 vision) by the age of 60.

In Nanoscope's pivotal RESTORE phase 2b trial, MCO-010 met its best corrected visual acuity (BCVA) primary endpoints in both dose groups at 52 weeks versus sham-control, with vision gains from baseline of >0.3 LogMAR, a result equivalent to three or more lines on an eye chart. In ongoing long-term follow-up, visual acuity gains were observed through 3 years, with no serious adverse events reported in treated eyes. Further multi-year follow up is scheduled with these patients to evaluate longer-term efficacy and safety.

For more information on MCO-010 and Nanoscope Therapeutics, visit: www.nanostherapeutics.com.