Nanoscope Therapeutics reports that it has initiated a rolling submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for MCO-010, a gene-agnostic optogenetic therapy for retinitis pigmentosa (RP). The submission marks the first BLA for a gene-agnostic therapy in a retinal disease, the company said in a press release. The FDA granted the application fast-track designation and is allowing a rolling review. Nanoscope expects to complete the BLA submission in early 2026.

Unlike traditional gene therapies, MCO-010 does not require identification of the patient’s genetic mutation. The therapy is administered through a one-time intravitreal injection and is designed to activate bipolar cells to restore light sensitivity after photoreceptor loss. In the phase 2b RESTORE trial, MCO-010 met its primary visual acuity endpoints at 1 year, with treated patients showing a gain of more than 3 lines on the eye chart compared to sham. Visual gains were maintained at 3 years without serious adverse events in the treated eyes.

Retinitis pigmentosa affects more than 100,000 people in the United States, with more than 25,000 legally blind. If approved, MCO-010 would be the first approved therapy to restore vision in these patients. The company has also completed the STARLIGHT phase 2 trial of MCO-010 in Stargardt disease patients. MCO-010 has received FDA fast-track designation and FDA orphan drug designation for both RP and Stargardt disease.