Nanoscope Therapeutics Inc., a clinical-stage biotechnology company, has recently announced that one of its drugs is approved for Phase 2b clinical trial. This drug, MCO-010, is a light activated optogenetic therapy that can help restore vision in patients with advanced retinitis pigmentosa (RP) regardless of the mutation that causes it. The study trial for the MCO-010 treatment will be a single injection into the eye in an office setting to confirm its ability to improve vision. This MCO gene therapy uses AAV2 vectors to target it to retinal cells. This gene therapy reprograms the retinal cells to make them photosensitive which can help restore vision. So far in the Phase 1/2a trial, MCO-010 has been shown to be safe for patients and to improve quality of life. This 2b trial starts in June and will be a randomized, double-blind, sham-controlled, multi-centered study in the United States. Patients with advanced RP will be enrolled.
What this means for Usher syndrome: Regardless of the mutation that causes retinitis pigmentosa, this optogenetic therapy has the potential to restore some vision. Usher syndrome vision loss is a type of RP. Depending on how the Phase 2b clinical trial goes, this MCO-010 treatment could be a potential therapy for individuals with vision loss caused by Usher syndrome in the future.