Nanoscope Therapeutics has secured significant regulatory milestones for its optogenetic therapy MCO-010 (sonpiretigene isteparvovec), receiving FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Stargardt disease and five Orphan designations from the European Medicines Agency covering multiple retinal dystrophies.
Regulatory Achievements Expand Global Development Pathways
The FDA's RMAT designation for Stargardt disease builds on MCO-010's existing regulatory recognitions, including prior Orphan Drug and Fast Track designations for both retinitis pigmentosa and Stargardt disease. In the United States, these combined designations provide opportunities for accelerated development and review, flexibility in trial design, and seven years of market exclusivity upon approval.
The EMA Orphan designations encompass several categories of retinal dystrophies, including non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies. This broad coverage has the potential to provide disease-agnostic regulatory paths forward for dozens of retinal conditions. In the European Union, EMA Orphan status provides opportunities for protocol assistance and up to 10 years of market exclusivity following approval.
"Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for SD and RP is a major validation for our therapies that warrant expedited development and review," said Sulagna Bhattacharya, CEO of Nanoscope. "Combined with 5 EMA Orphan designations, these achievements highlight the global momentum behind our MCO platform as a potential vision-restoring therapy for patients with few or no treatment options."
Disease-Agnostic Optogenetic Platform
MCO (multi-characteristic opsin) is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including retinitis pigmentosa, Stargardt disease, and geographic atrophy. The therapy works by activating highly dense bipolar retinal cells to become light sensitive and employing the remaining visual circuitry following photoreceptor death.
Notably, MCO does not require genetic testing, surgical intervention, or repeat dosing, which may enable broad patient applicability within existing retina office workflows. This approach addresses a significant unmet need, as retinal degenerative diseases affect millions of patients globally and are life-altering, chronically debilitating conditions that lack broadly effective treatment options.
Clinical Progress Across Multiple Indications
Nanoscope has demonstrated positive results in the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (NCT04945772). Following these results, the company has initiated a rolling Biologics License Application (BLA) submission to the FDA. If approved, MCO-010 has the potential to be the standard of care for retinitis pigmentosa patients, administered as a one-time, in-office injection.
The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025. Additionally, a Phase 2 program for MCO in geographic atrophy is expected to start by the end of 2025, with other IND-ready programs including Leber congenital amaurosis.
