Nanoscope Therapeutics announced positive top-line results from a Phase 2b trial of MCO-010 for retinitis pigmentosa (RP). The trial demonstrated significant improvements in visual function for patients treated with MCO-010, with 88.9% showing clinically meaningful improvements in mobility and shape discrimination tests. Additionally, the treatment was well-tolerated with no severe adverse events. This optogenetic therapy offers hope for patients with RP, a condition currently lacking effective treatments. Nanoscope plans to engage with the FDA and other regulatory agencies to expedite the availability of this potential therapy.
What this means for Usher syndrome: Since treatment with MCO-010 is gene agnostic, which means it does not target specific genetic mutations but instead aims to restore vision by activating remaining retinal cells, this approach is potentially beneficial for all types of USH.
