Nanoscope Therapeutics Inc. has initiated a rolling submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for its investigational gene-agnostic therapy, MCO-010, which is intended for the treatment of severe vision loss in retinitis pigmentosa (RP) patients.

The optogenetic therapy is administered via a single, in-office intravitreal injection. It is designed to bypass the need for viable photoreceptors and resensitize bipolar retinal cells to light. The therapy does not require surgical intervention, repeat dosing, or genetic testing, and offers broad applicability to RP patients regardless of genetic subtype. The gene-agnosticism component is significant because RP is linked to more than 100 genes and 1,000 mutations.

MCO-010’s efficacy was demonstrated in the RESTORE phase 2b trial—a randomized, double-masked, sham-controlled study. At 52 weeks compared with controls, patients who received MCO-010 in both dose groups met the primary BCVA endpoint and achieved a vision gain of 3 or more lines on an eye chart. Long-term follow-up indicated sustained visual gains through 3 years posttreatment. No serious adverse events were reported in treated eyes.

The company is continuing to monitor long-term safety and efficacy and is planning a phase 3 registrational trial this year.

In addition to RP, MCO-010 has shown promise in the STARLIGHT phase 2 study for Stargardt disease. Preclinical development is also under way for Leber congenital amaurosis and geographic atrophy.

RP affects over 100,000 individuals in the US: the average RP patient loses one line of vision every 3 years, and most become legally blind by age 60. More than 25,000 patients are classified as legally blind from the condition.

The FDA has granted MCO-010 fast-track designation and is reviewing the BLA under a rolling submission framework. Full completion is expected in early 2026.

MCO-010 has also been granted Orphan Drug designation for RP and Stargardt disease.