Nanoscope Therapeutics Announces PMDA Acceptance and Priority Review of New Drug Application for MOGENRY® in Japan for the Treatment of Inherited Retinal Dystrophies

• If approved, MOGENRY® would be the first disease-agnostic treatment for patients with inherited retinal dystrophies (IRDs)
• PMDA has accepted the application and the file will be reviewed on Priority, building on the Sakigake and Orphan drug designations granted to MOGENRY for IRDs in Japan
• Application is supported by positive RESTORE Phase 2b/3 and long-term follow-up data in Retinitis Pigmentosa and STARLIGHT Phase 2 data in Stargardt disease

DALLAS, Oct. 1, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc. ("Nanoscope"), a biotechnology company focused on developing and commercializing novel, disease–agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that Japan's Pharmaceuticals and Medical Devices Agency (PMDA) has accepted for review, and granted priority review to, the Company's New Drug Application (NDA) seeking marketing approval from the Ministry of Health, Labour and Welfare (MHLW) of MOGENRY^®(sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration, for the treatment of inherited retinal dystrophies (IRDs).

Priority review reflects MOGENRY's designation under the Sakigake Designation System, Japan's expedited regulatory pathway for innovative medical products, and is intended to shorten the time to a regulatory decision. Separately, the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's Biologics License Application (BLA) for MOGENRY for the treatment of retinitis pigmentosa (RP) with severe vision loss, which remains under review.

"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," said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope Therapeutics. "With MOGENRY now under priority review in Japan, with potential approval in both Japan and the United States expected in the first half of 2027, we are advancing our goal of making a one-time, disease-agnostic vision-restoring therapy available to patients across major markets. The strength of our RESTORE data and the durability observed in long-term follow-up in the REMAIN study reinforce our confidence in MOGENRY's potential, and we look forward to working closely with the PMDA and MHLW throughout the review process."

The application is supported by positive efficacy and safety data from the Phase 1/2a trial in RP (NCT04919473), the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in RP (NCT04945772), and the STARLIGHT multicenter, Phase 2 trial in Stargardt disease (NCT05417126). RESTORE met its primary and key secondary endpoints, demonstrating improvements in visual acuity at weeks 52 and 76. MOGENRY was well tolerated, with no treatment-related serious adverse events reported. Most patients dosed in RESTORE have continued into the REMAIN study, which provides long-term follow-up data included in the submission.

"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," said Samarendra Mohanty, Ph.D., Co-Founder, President and Chief Scientific Officer of Nanoscope Therapeutics. "We are very encouraged by the continued constructive and collaborative dialogue with the Japanese regulators over the past 1 year. Seeing the PMDA accept our NDA across IRDs and grant it priority review, on the strength of MCO-010's unique mechanism of action and clinical evidence, is incredibly exciting for our entire team. It reflects our conviction that vision restoration does not have to be limited to a single gene or a single disease, and we are proud to be one step closer to bringing MOGENRY to IRD patients who have been waiting for a treatment. Each regulatory milestone renews our commitment to moving with urgency for people living with IRDs worldwide."

If approved, MOGENRY would become the first disease-agnostic therapy in Japan to improve vision in patients with IRDs, offering a one-time, in-office treatment that does not require genetic testing or administration in a surgical suite.

About Inherited Retinal Dystrophies
Inherited retinal dystrophies (IRDs) are a genetically diverse group of rare disorders, caused by mutations in more than 250 genes, in which progressive loss of photoreceptor cells leads to impaired vision and, in most cases, complete blindness. IRDs include retinitis pigmentosa (RP), the most common IRD, as well as Stargardt disease, cone-rod dystrophies, and Leber congenital amaurosis, among others. Because gene-specific therapies address only a single genetic cause, the vast majority of patients with IRDs have no approved treatment option. In Japan, IRD is one of the leading causes of visual impairment, and RP is designated as an intractable disease.

About MOGENRY (sonpiretigene isteparvovec; MCO-010)
Nanoscope's multi-characteristic opsin (MCO) technology utilizes a proprietary genetically engineered synthetic opsin to enable optimized performance in terms of high light sensitivity across a broad spectrum and fast kinetics. After Nanoscope's Co-Founder, President, and Chief Scientific Officer, Samarendra Mohanty, envisioned and invented the technology, his team has spent over a decade evaluating its performance across multiple animal models and human diseases. MOGENRY is an investigational, one-time, in-office, intravitreal optogenetic gene therapy built on Nanoscope's MCO platform. By delivering a multi-characteristic opsin gene to the highly dense bipolar retinal cells, MOGENRY makes these surviving cells directly light-sensitive, enabling them to utilize the remaining visual circuitry following photoreceptor loss. MOGENRY does not require genetic testing, invasive surgery, or repeat dosing, and is designed for administration within existing retina office workflows. MOGENRY is under review by the FDA for the treatment of RP with severe vision loss and under priority review by the PMDA and MHLW for the treatment of IRDs in Japan.

About Nanoscope Therapeutics
Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the FDA has accepted and filed the Company's BLA for MOGENRY, and the PMDA has accepted for priority review the Company's NDA for MOGENRY for the treatment of IRDs in Japan. If approved, MOGENRY has the potential to become the standard of care for patients with RP and other IRDs, administered as a one-time, in-office injection without the need for genetic testing. The Company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations covering non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. MCO-010 has also received Sakigake and Orphan designations for inherited retinal dystrophies (IRDs) in Japan and an Orphan designation for IRDs in Saudi Arabia. A Phase 2 program for MCO in geographic atrophy is expected to start in 2026, and other IND-ready programs include Leber congenital amaurosis (LCA). Learn more at NanosTherapeutics.com and follow @NSTherapeutics on X and on LinkedIn.

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Correction: The first quote has been updated.

SOURCE Nanoscope Therapeutics