Japan’s Ministry of Health, Labour and Welfare has granted Sakigake and Orphan Drug designations to MCO-010, an optogenetic gene therapy in development by Nanoscope Therapeutics for patients with severe vision loss from inherited retinal diseases. According to the company, MCO-010 is the first retinal gene therapy to receive both designations in Japan.

Sakigake is Japan’s accelerated review program for innovative therapies addressing high unmet medical need and is broadly comparable to the US Food and Drug Administration’s Breakthrough Therapy, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations, all of which MCO-010 has previously received. 

Nanoscope also reported that regulatory agencies in the United States, European Union, and Japan have conditionally approved the brand name Mogenry for MCO-010, which is designed as a one-time intravitreal therapy intended to restore light sensitivity by targeting downstream retinal cells in patients with advanced photoreceptor loss. 

Nanoscope has initiated a rolling submission of a biologics license application to the FDA for MCO-010 in retinitis pigmentosa and is pursuing development in Stargardt disease, with a phase 3 registrational trial planned for early 2026. A phase 2 program in geographic atrophy is also planned for early 2026.