Nanoscope Secures Japan MHLW Sakigake and Orphan Drug Designations Across Inherited Retinal Diseases - A First for a Retinal Gene Therapy

• Japan's Ministry of Health, Labor and Welfare (MHLW) grants MCO-010 Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations, enabling an accelerated regulatory pathway to treat patients with severe vision loss from inherited retinal diseases
• Builds on strong global regulatory momentum, including five EMA Orphan designations and multiple FDA expedited programs across retinal indications
• FDA, EMA and PMDA conditionally approve MOGENRY™ brand name for MCO-010 as Nanoscope's rolling BLA with the FDA for retinitis pigmentosa advances
• Positions MCO-010 for global pre-commercialization readiness across major markets, reinforcing Nanoscope's leadership in optogenetic therapy

DALLAS, Jan. 20, 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 Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Sakigake and Orphan Drug designations to MCO-010, Nanoscope's lead asset to treat patients with severe vision loss from inherited retinal diseases (IRDs).

The Sakigake designation, Japan's premier fast-track regulatory program for innovative therapies addressing serious unmet medical needs, is designed to accelerate patient access to breakthrough treatments. Sakigake status enables prioritized regulatory consultation and review—often targeting a six-month review timeline—through close collaboration with the Pharmaceuticals and Medical Devices Agency (PMDA) and may support premium pricing following approval. Combined with Orphan Drug designation (for use in less than 50,000 patients with high medical need), the decision establishes a strong regulatory pathway for MCO-010 in Japan. MCO-010 is the first ever retinal gene therapy to be granted both Sakigake and Orphan Drug designations.

"These designations from Japan's MHLW mark another pivotal milestone for Nanoscope," said Samarendra Mohanty, PhD, President, and Chief Scientific Officer of Nanoscope Therapeutics. "Sakigake recognition underscores the novelty of MCO-010 as a one-time, in-office intravitreal optogenetic therapy and its potential to address profound vision loss where no approved treatments exist. Together with our progress in the U.S. and Europe, we are advancing one of the most comprehensive global regulatory strategies in optogenetic retinal gene therapy for patients living with inherited retinal diseases."

Strong, Major Market Regulatory Momentum

The MHLW's decision builds on accelerated regulatory pathways across other major markets:

• European Union (EMA): Five Orphan designations spanning non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies
• United States (FDA):
• Orphan Drug and Fast Track designations for retinitis pigmentosa (RP)
• Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations for Stargardt disease (SD)

In addition, the FDA, EMA and PMDA have conditionally approved the brand name MOGENRY™ for MCO-010, while Nanoscope continues to advance its rolling Biologics License Application (BLA) for marketing approval for RP patients in the United States.

With these regulatory pathways now established in Japan, the United States, and Europe, MCO-010 represents the most advanced optogenetic platform therapy in development worldwide. These coordinated designations support accelerated development timelines, enhanced regulatory engagement, and the opportunity to be the first optogenetic therapy approved across major markets.

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 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 early 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. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start in early 2026. Other IND-ready programs include Leber congenital amaurosis (LCA).

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

SOURCE Nanoscope Therapeutics