Dallas – October 01, 2026 -- Japan's Pharmaceuticals and Medical Devices Agency (PMDA) has accepted and granted priority review to Nanoscope Therapeutics' New Drug Application for MOGENRY (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy for inherited retinal dystrophies (IRDs). If approved by the Ministry of Health, Labour and Welfare, MOGENRY would become the first disease-agnostic treatment available to IRD patients in Japan, a condition caused by mutations across more than 250 genes that leads to progressive photoreceptor loss and blindness.
PMDA priority review builds on prior Sakigake and Orphan Drug designations
The priority review status reflects MOGENRY's standing under Japan's Sakigake Designation System, an expedited regulatory pathway for innovative medical products, and is designed to shorten the time to a regulatory decision. MOGENRY had already secured Sakigake and Orphan Drug designations for IRDs in Japan prior to this NDA acceptance.
FDA review of U.S. Biologics License Application runs in parallel
The U.S. Food and Drug Administration has separately accepted and filed Nanoscope's Biologics License Application for MOGENRY targeting retinitis pigmentosa (RP) with severe vision loss, which remains under review. CEO Sulagna Bhattacharya said potential approvals in both Japan and the United States are expected in the first half of 2027.
Clinical package spans three trials in RP and Stargardt disease
The Japanese application is supported by data from the Phase 1/2a trial in RP (NCT04919473), the RESTORE Phase 2b/3 randomized, double-masked, sham-controlled trial in RP (NCT04945772), and the STARLIGHT Phase 2 trial in Stargardt disease (NCT05417126). RESTORE met its primary and key secondary endpoints, showing improvements in visual acuity at weeks 52 and 76, with no treatment-related serious adverse events reported. Most RESTORE participants have continued into the REMAIN long-term follow-up study, whose durability data were included in the submission.
Therapy targets downstream retinal cells to bypass genetic diagnosis requirement
MOGENRY is designed to act downstream of the specific genetic defect by delivering a multi-characteristic opsin gene to surviving bipolar retinal cells, making them directly light-sensitive. Co-Founder and Chief Scientific Officer Samarendra Mohanty said the approach allows a single therapy to restore vision in ambient lighting regardless of which of the hundreds of IRD-linked genes is responsible. The treatment is administered as a one-time, in-office intravitreal injection that requires no genetic testing, surgical suite, or repeat dosing.
IRDs remain a leading cause of visual impairment in Japan with no approved options
Retinitis pigmentosa, the most common IRD, is designated an intractable disease in Japan, where IRDs are a leading cause of visual impairment. Because gene-specific therapies address only single genetic causes, the majority of IRD patients currently have no approved treatment option.