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

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Nanoscope Therapeutics

21 Jan, 2026, 06:00 CST

  • Japan's Ministry of Health, Labour and Welfare (MHLW) grants MCO-010 Sakigake (Pioneering Regenerative Medical Product) and Orphan Drug designations, enabling an accelerated regulatory pathway for patients with severe inherited retinal diseases
  • Enhances strong global regulatory momentum, including multiple FDA expedited programs and five EMA Orphan designations
  • FDA, EMA and PMDA conditionally approve the MOGENRY™ brand name
  • Strengthens global pre-commercialization readiness and reinforces leadership in optogenetic therapy

DALLAS, Jan. 21, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a biotechnology company developing disease-agnostic therapies for vision loss caused by retinal degeneration, today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted both Sakigake (Pioneering Regenerative Medical Product) and Orphan Drug designations to MCO-010, its lead optogenetic therapy for patients with severe vision loss from inherited retinal dystrophies (IRDs). Approximately 50,000 patients in Japan are affected by IRDs.

Sakigake is Japan's premier fast-track regulatory program for innovative therapies addressing serious unmet medical needs. The designation enables prioritized regulatory consultation and review—often targeting a six-month review timeline—through collaboration with the Pharmaceuticals and Medical Devices Agency (PMDA) and may support premium pricing following approval. Combined with Orphan Drug designation for rare, high-need conditions, the decision establishes a clear regulatory pathway for MCO-010 in Japan and marks the first Sakigake designation granted to a retinal gene therapy.

"These designations represent an important milestone for Nanoscope and for patients living with inherited retinal diseases," said Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope Therapeutics. "Sakigake recognition underscores the novelty of MCO-010 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."

The MHLW decision builds on strong regulatory momentum across major markets, including five Orphan designations from the European Medicines Agency (EMA) and multiple expedited programs from the U.S. Food and Drug Administration (FDA) across retinal indications. In addition, the FDA, EMA and PMDA have conditionally approved the MOGENRY™ brand name, as Nanoscope continues to advance its rolling Biologics License Application (BLA) for retinitis pigmentosa in the United States.

With regulatory pathways now established in Japan, the United States, and Europe, MCO-010 is positioned as the most advanced optogenetic platform therapy in development worldwide.

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

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SOURCE Nanoscope Therapeutics