The race to restore sight for patients living with retinal degeneration just gained serious momentum.
Nanoscope Therapeutics (Texas, United States) recently announced that its lead candidate MCO-010 (sonpiretigene isteparvovec) has earned five new Orphan designations from the European Medicines Agency (EMA), covering a wide range of retinal dystrophies. At the same time, the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Stargardt disease (SD)—a strong signal of regulatory confidence in the therapy’s potential.
The EMA designations span non-syndromic and syndromic rod-dominant dystrophies, cone-dominant dystrophies and macular dystrophies, paving a pathway that could impact dozens of currently untreatable conditions. The FDA’s RMAT status for SD builds on prior Orphan Drug and Fast Track designations in both SD and retinitis pigmentosa (RP).
READ MORE: FDA Grants Fast Track Designation to Oral Treatment for Retinitis Pigmentosa
These twin recognitions mark a critical step in positioning MCO-010 as a potential disease-agnostic therapy platform for patients living with progressive vision loss.
Nanoscope Therapeutics reveals the big news on LinkedIn.
“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, in a news release. “Combined with five 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.”
READ MORE: Nanoscope Kickstarts BLA Submission for Restorative RP Therapy, Adds to Advisory Board
A vision-restoring platform
At its core, MCO-010 (multi-characteristic opsin) optogenetic platform is designed to reimagine how degenerative retinal diseases are treated. Unlike gene-specific or surgical interventions, MCO-010 is a one-time, in-office intravitreal therapy.
It works by activating dense bipolar retinal cells to become light-sensitive, harnessing existing visual circuitry even after photoreceptor death. The approach requires no genetic testing, no surgery and no repeat dosing, making it broadly compatible with standard retina clinic workflows.
Clinical progress has reinforced this promise. In the RESTORE Phase IIb/III trial for retinitis pigmentosa (RP)—a multicenter, randomized, double-masked, sham-controlled study—MCO-010 delivered positive results, prompting Nanoscope to initiate a rolling Biologics License Application (BLA) submission to the FDA. If approved, the therapy could become a new standard of care for RP patients.
Momentum extends beyond RP. The STARLIGHT Phase II trial in SD yielded encouraging data, and a Phase III registrational trial in SD is set to launch in 2025. In parallel, Nanoscope plans a Phase II trial in geographic atrophy (GA) later this year, further expanding the therapy’s potential reach.
READ MORE: FDA Grants Breakthrough Therapy Designation to Belite Bio’s Tinlarebant for Stargardt Disease
A step forward for patients
Retinal degenerative diseases such as RP, SD, macular dystrophies and syndromic retinal disorders affect millions globally, with no effective therapies available today.
Regulatory recognition creates new opportunities. In Europe, EMA Orphan status offers protocol assistance and up to 10 years of market exclusivity following approval. In the U.S., Orphan, Fast Track and RMAT designations enable accelerated development, flexible trial design and seven years of exclusivity upon approval.
Together, these regulatory milestones combined with advancing clinical programs underscore the potential of MCO-010 to transform the treatment landscape for retinal degeneration and offer hope to patients facing irreversible vision loss.
Editor’s Note: Read the official press release for further information. Results from the RESTORE Phase 2b/3 for RP (NCT04945772) and STARLIGHT Phase 2 clinical trial for SD (NCT05417126) can be found at clinicaltrials.gov.
This content is intended exclusively for healthcare professionals. It is not intended for the general public. Products or therapies discussed may not be registered or approved in all jurisdictions, including Singapore.