Key Takeaways

  • Japan’s PMDA has accepted Nanoscope Therapeutics’ NDA for Mogenry and granted the therapy priority review for inherited retinal dystrophies
  • The application is supported by clinical data from the RESTORE, STARLIGHT, and earlier retinitis pigmentosa studies, along with long-term follow-up from REMAIN
  • Mogenry is also under FDA review in the United States for retinitis pigmentosa with severe vision loss, with Nanoscope anticipating possible regulatory decisions in both markets in the first half of 2027

Japan’s Pharmaceuticals and Medical Devices Agency has accepted Nanoscope Therapeutics’ new drug application for Mogenry (sonpiretigene isteparvovec, MCO-010) and granted the optogenetic gene therapy priority review for the treatment of inherited retinal dystrophies.

The application seeks marketing approval from Japan’s Ministry of Health, Labour and Welfare. Priority review follows Mogenry’s Sakigake designation, an expedited pathway for innovative therapies intended to accelerate regulatory review in Japan.

Mogenry is designed as a disease-agnostic optogenetic therapy for patients who have experienced photoreceptor loss from retinal degeneration. Rather than targeting a specific pathogenic gene, the therapy delivers a multi-characteristic opsin gene intended to make surviving retinal bipolar cells light-sensitive and thereby use remaining retinal circuitry to restore visual function.

The Japanese submission is supported by data from a phase 1/2a study in retinitis pigmentosa, the phase 2b/3 RESTORE trial in retinitis pigmentosa, and the phase 2 STARLIGHT trial in Stargardt disease. According to Nanoscope, RESTORE met its primary and key secondary endpoints, with improvements in visual acuity reported at weeks 52 and 76. No treatment-related serious adverse events were reported.

Most patients treated in RESTORE subsequently entered the REMAIN long-term follow-up study, and those durability data were also included in the Japanese submission. Nanoscope has separately reported continued long-term follow-up from the RESTORE program, including 4-year data presented in 2026.

“The PMDA's acceptance of our application with priority review is a pivotal moment for Nanoscope and for the many patients living with IRDs who have no approved treatment options today,” Sulagna Bhattacharya, chief executive officer of Nanoscope Therapeutics, said in the company’s announcement.

Mr. Bhattacharya added that the company expects potential regulatory decisions in Japan and the United States during the first half of 2027, although those timelines remain subject to regulatory review.

Mogenry is also under FDA review in the United States. In September 2026, the agency accepted and filed Nanoscope’s Biologics License Application for the treatment of retinitis pigmentosa with severe vision loss.

Nanoscope said the Japanese indication is broader, covering inherited retinal dystrophies rather than retinitis pigmentosa alone.

“We designed MCO-010 to act downstream of the genetic defect, so that a single therapy could restore vision in ambient lighting conditions regardless of which of the hundreds of genes is driving a patient's disease,” Samarendra Mohanty, PhD, co-founder, president, and chief scientific officer of Nanoscope Therapeutics, said.

If approved in Japan, Mogenry would be administered as a one-time, in-office treatment and would not require genetic testing or administration in a surgical suite, according to the company. Nanoscope also said it would be the first disease-agnostic therapy approved in Japan to improve vision in patients with inherited retinal dystrophies.