Nanoscope Secures EMA Orphan Designations and FDA RMAT Status for Retinal Gene Therapy MCO-010
Nanoscope Therapeutics announced regulatory milestones in both Europe and the United States for its lead gene therapy candidate, MCO-010 (sonpiretigene isteparvovec).
The European Medicines Agency (EMA) has granted Orphan Drug designation for MCO-010 across five categories of retinal dystrophies, including non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies. Meanwhile, the FDA has awarded Regenerative Medicine Advanced Therapy (RMAT) designation for MCO-010 in Stargardt disease (SD).
The EMA designations offer a potential broad regulatory framework that spans dozens of retinal conditions, reinforcing MCO-010’s positioning as a disease-agnostic therapy. In the EU, Orphan Drug designation includes protocol assistance and up to 10 years of market exclusivity post-approval.
The new FDA RMAT designation for Stargardt disease builds upon earlier Orphan Drug and Fast Track designations for both Stargardt disease and retinitis pigmentosa (RP). In the U.S., Orphan, Fast Track, and RMAT statuses provide accelerated development pathways, trial design flexibility, and 7 years of market exclusivity upon approval.
The multi-characteristic opsin (MCO) platform is a one-time, in-office intravitreal gene therapy designed to restore vision in patients with photoreceptor degeneration, including RP, SD, and geographic atrophy (GA). Unlike other gene therapies, MCO does not require genetic testing, surgical delivery, or repeat dosing, according to Nanoscope.
MCO-010 works by activating bipolar retinal cells—abundant even in advanced retinal disease—to become light-sensitive, leveraging the surviving visual circuitry after photoreceptor loss. This approach enables streamlined integration into standard retina practice workflows.
Clinical Progress and Upcoming Trials
Nanoscope’s clinical programs include:
RP: Positive results reported from the phase 2b/3 RESTORE trial
Stargardt Disease: Encouraging findings from the Phase 2 STARLIGHT trial
Upcoming Trials: A phase 3 registrational trial in SD and a phase 2 trial in GA are expected to launch before the end of 2025