Nanoscope Therapeutics Secures Dual “Sakigake” and Orphan Designations in Japan

DALLAS —January 20, 2026— Nanoscope Therapeutics has achieved a historic regulatory milestone for its lead candidate, MCO-010 (conditionally branded as MOGENRY). Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted the therapy both Sakigake (Pioneering Regenerative Medical Product) and Orphan Drug designations, marking the first time a retinal AAV gene therapy has received both honors simultaneously.

Accelerating AAV-Mediated Vision Restoration in Japan

The Sakigake designation is Japan’s premier fast-track program for innovative therapies. This status provides Nanoscope with prioritized AAV regulatory consultation and an expedited review timeline—targeting a six-month window—through close collaboration with the PMDA. This AAV-based designation also supports premium pricing structures, recognizing the breakthrough nature of the AAV2-delivered optogenetic platform.

A Global AAV Optogenetic Powerhouse

The announcement in Japan builds on massive AAV regulatory momentum across major markets. MCO-010 is currently the most advanced AAV optogenetic platform globally, addressing photoreceptor loss in diseases like Retinitis Pigmentosa (RP) and Stargardt disease (SD). In the U.S., Nanoscope is advancing a rolling AAV Biologics License Application (BLA), while the EU has granted five separate AAV Orphan designations for the program.

The MOGENRY Advantage: Mutation-Agnostic AAV Healing

Unlike traditional AAV gene replacement therapies that target one specific mutation, MOGENRY™ is disease-agnostic. It uses a proprietary AAV2 vector to deliver an ambient-light sensitive opsin transgene into surviving bipolar cells.

• Universal AAV Delivery: The AAV payload works regardless of the patient’s underlying genetic mutation.

• Streamlined AAV Workflow: The AAV therapy is a one-time, in-office intravitreal injection, requiring no complex AAV surgical suite or pre-treatment genetic testing.

• Persistent AAV Expression: This AAV2 approach is designed for long-term transgene expression, functionalizing the inner retina to act as a new light sensor.