Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations

• European Medicines Agency grants MCO-010 five Orphan designations across a wide spectrum of retinal diseases involving photoreceptor loss, including syndromic and non-syndromic, rod- and cone-dominant, and macular dystrophies.
• FDA grants MCO-010 Regenerative Medicine Advanced Therapy (RMAT) designation, in addition to current Orphan Drug and Fast Track designations, for Stargardt disease.
• Nanoscope is redefining the standard of care for high unmet need retinal degenerations with its disease-agnostic MCO-010 optogenetic platform.

DALLAS, Sept. 2, 2025 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel disease-agnostic therapies for patients with photoreceptor loss and vision impairment from retinal degeneration, today announced that the European Medicines Agency (EMA) has granted Orphan designations to MCO-010 (sonpiretigene isteparvovec) across five categories of retinal dystrophies. In addition, the U.S. Food and Drug Administration (FDA) has granted RMAT designation for MCO-010 in Stargardt disease (SD).

The EMA Orphan designations cover non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies, providing a potential disease-agnostic regulatory path forward for dozens of retinal conditions. The new FDA RMAT designation for SD builds on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa (RP) and SD. The EMA and FDA actions underscore regulatory agency recognition of MCO-010's potential to address significant unmet needs in retinal degenerative diseases.

"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 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."

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 RP, SD, and geographic atrophy (GA). By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO does not require genetic testing, surgical intervention, or repeat dosing, enabling broad patient applicability within existing retina office workflows.

Nanoscope's vision-improving clinical programs are advancing globally, with positive Phase 2b/3 RESTORE trial results in RP and encouraging findings from the Phase 2 STARLIGHT trial in SD. A Phase 3 registrational trial in SD and a Phase 2 trial in GA are expected to begin by the end of 2025.

Retinal degenerative diseases, including RP, SD, macular dystrophies, and syndromic retinal disorders, affect millions of patients globally. These diseases are life-altering, chronically debilitating, and lack broadly effective treatment options. In the European Union, EMA Orphan status provides opportunities for protocol assistance and up to 10 years of market exclusivity following approval. In the United States, Orphan, Fast Track, and RMAT designations provide opportunities for accelerated development and review, flexibility in trial design, and seven years of market exclusivity upon approval.

About Nanoscope Therapeutics

Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2025. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start by the end of 2025. Other IND-ready programs include Leber congenital amaurosis (LCA).

Contact:
Nanoscope Therapeutics
(817) 857-1186
[email protected]

SOURCE Nanoscope Therapeutics