Nanoscope Begins Rolling BLA Submission to FDA for Seeking Approval for its Optogenetic Therapy

Nanoscope Therapeutics, a developer of therapies for retinal diseases, has submitted the first modules of a Biologics License Application (BLA) to the US Food & Drug Administration (FDA) for MCO-010, its emerging optogenetic therapy for restoring some vision to people with advanced retinitis pigmentosa (RP). The FDA’s approval of the BLA would make MCO-010 available to advanced RP patients in the US. MCO-010 is designed to work regardless of the mutated gene causing RP.

The BLA modules are being submitted under rolling review, meaning the modules are submitted as they are completed (i.e., not all at once). Nanoscope anticipates that BLA module submission will be completed in early 2026.

MCO-010 has also received Fast Track designation from the FDA which can make it eligible for priority (expedited) review. The Fast Track designation can be given to therapies for serious, unmet medical conditions.

MCO-010 is designed for people who have lost most or all their photoreceptors, the light-sensing retinal cells that make vision possible. The treatment, a small drop of liquid delivered by a one-time intravitreal injection, uses a human-engineered virus to deliver copies of a Multi-Characteristic Opsin (MCO) gene to bipolar cells which normally don’t sense light but often survive after photoreceptors are lost to advanced retinal disease. The company says that MCO-010 is enabling the recipient’s bipolar cells to sense ambient light, thereby working as a backup system for lost photoreceptors. The approach doesn’t use goggles or eyewear to enhance the visual information coming into the retina.

In March 2024, Nanoscope announced that it met the primary and secondary endpoints for vision improvements in its RESTORE Phase 2b clinical trial for MCO-010. A total of 27 patients were enrolled in the trial. Nine patients received a high-dose of MCO-010, nine received a low-dose, and nine received sham/control.

Nanoscope is planning a Phase 3 clinical trial of MCO-010 for people with Stargardt disease.