Back in October 2024, Nanoscope Therapeutics announced plans to begin a rolling FDA submission for its potential treatment MCO-010, following a positive meeting with the agency. Less than a year later, that plan is becoming reality. In July 2025, Nanoscope officially started its Biologics License Application (BLA) for MCO-010, a gene-agnostic optogenetic therapy designed to help people with retinitis pigmentosa (RP), including those with Usher syndrome, regain some ability to see light and shapes.
Over the last few months, Nanoscope has released additional press releases that higllighted recent study results show lasting benefits. In the REMAIN study, people with advanced RP retained meaningful vision improvements for three years after just one MCO-010 injection, with no serious side effects. A separate five-year safety study confirmed the treatment remained safe and stable over time. These findings suggest MCO-010 could bring long-term vision improvements for people who currently have no treatment options.
MCO-010 is also showing promise beyond RP. In the STARLIGHT study, people with Stargardt disease, another inherited retinal condition, experienced measurable vision improvements and no major safety issues. Because MCO-010 works without targeting a specific gene, it may one day help a wide range of retinal conditions, including Usher syndrome.
What this means for the Usher syndrome community: For people and families affected by Usher syndrome, these results bring hope. Since MCO-010 doesn’t rely on fixing one specific gene, it could one day help those with different or unknown mutations who have never had treatment options. While the FDA review continues, Nanoscope’s continued progress marks a major step toward restoring sight and possibly independence for those living with both hearing and vision loss.
