DALLAS, March 30, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that it has received Orphan Drug Designation in the Kingdom of Saudi Arabia for its lead therapy, MCO-010, for the treatment of patients with inherited retinal dystrophies (IRDs) having severe vision loss.
The broad designation from Saudi regulatory authorities further strengthens Nanoscope's expanding global regulatory footprint and reflects increasing international recognition of the company's proprietary Multi-Characteristic Opsin (MCO) platform as a potential first-in-class, disease-agnostic optogenetic therapy for vision restoration.
"MCO-010 development as a disease-agnostic optogenetic therapy continues to progress with regulatory authorities worldwide," said Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope Therapeutics. "With this designation in Saudi Arabia, we are further accelerating our efforts to bring this therapy to patients globally, including in regions where access to advanced retinal treatments has historically been limited."
Building a Global Regulatory Pathway Across Major Markets
The Saudi designation builds on a series of significant regulatory milestones across the United States, Japan, and Europe, positioning MCO-010 among the most advanced optogenetic therapies in development globally.
In the United States, MCO-010 has received Orphan Drug and Fast Track designations for retinitis pigmentosa, as well as Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations for Stargardt disease. Nanoscope has also initiated a rolling Biologics License Application (BLA) submission for retinitis pigmentosa, underscoring the therapy's late-stage development progress.
In Japan, the Ministry of Health, Labour and Welfare (MHLW) granted both Sakigake (Pioneering Regenerative Medical Product) and Orphan Drug designations for IRDs to MCO-010—marking the first time a retinal gene therapy has received such broad IRD designations in the country. These designations provide a clear accelerated regulatory pathway, including prioritized consultation and the potential for expedited review timelines.
Across Europe, MCO-010 has received five Orphan designations from the European Medicines Agency (EMA), spanning a broad range of IRDs, supporting a disease-agnostic regulatory strategy across multiple indications.
IRDs are a group of rare, progressive diseases that lead to severe vision loss or blindness and currently lack treatment options. By progressing regulatory pathways across North America, Europe, Asia, and now the Middle East, Nanoscope is advancing one of the most comprehensive global development strategies in optogenetic therapy.
About the MCO Platform
MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including Retinitis Pigmentosa (RP), Stargardt disease (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 treatment does not require genetic testing, invasive surgery, or repeat dosing, enabling broad patient applicability within existing retina office workflows.
About Nanoscope Therapeutics
Nanoscope Therapeutics is developing a 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 is ongoing. 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 2026. 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. MCO-010 has also received Sakigake (Pioneer) and Orphan Drug designations in Japan from MHLW and Orphan Drug designation in the Kingdom of Saudi Arabia. A Phase 2 program for MCO in geographic atrophy (GA) is also expected to start in 2026. Other IND-ready programs include Leber congenital amaurosis (LCA).
Contact:
Nanoscope Therapeutics, Inc
+1 (817) 857-1186
PR@nanostherapeutics.com
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