Nanoscope Therapeutics has achieved significant regulatory milestones for its optogenetic therapy candidate MCO-010 (sonpiretigene isteparvovec), paving the way for accelerated development and broader disease applications.
The European Medicines Agency has granted orphan drug designations for MCO-010 in five categories of retinal dystrophies, including non-syndromic and syndromic rod-dominant and cone-dominant disorders, as well as macular dystrophies, offering a potential disease-agnostic regulatory route for numerous retinal conditions, according to the company.
In the United States, the Food and Drug Administration has awarded Regenerative Medicine Advanced Therapy (RMAT) designation for MCO-010 in Stargardt disease. This builds upon its existing orphan drug and Fast Track designations for both Stargardt disease and retinitis pigmentosa, the company said.
MCO-010 is a one-time, in-office intravitreal optogenetic therapy designed to restore vision in patients with photoreceptor degeneration—such as retinitis pigmentosa, Stargardt disease and geographic atrophy—by activating bipolar retinal cells to become light-sensitive. The company claims this platform does not require genetic testing, surgical delivery or repeat dosing, allowing for integration into existing retina practice workflows.
Positive results have been reported from the Phase 2b/3 RESTORE trial in retinitis pigmentosa and the Phase 2 STARLIGHT trial in Stargardt disease. A Phase 3 registrational study in Stargardt disease and a Phase 2 trial in geographic atrophy are expected to commence by the end of 2025.
