Nanoscope’s gene therapy, MCO-010 receives Sakigake and Orphan Drug Designations in Japan

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for MCO-010 from Japan’s Ministry of Health, Labour and Welfare (MHLW), making it the first retinal gene therapy to do so in Japan.

Sakigake designation is Japan’s “premier fast-track regulatory program for innovative therapies addressing serious unmet medical needs, designed to accelerate patient access to breakthrough treatments.”¹ According to the company, the designation also allows for prioritized regulatory consultation and review.

MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including retinitis pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). The company states that by activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death.

Samarendra Mohanty, PhD, president and chief scientific officer of Nanoscope Therapeutics, commented on the designations in a press release from the company, saying, "These designations from Japan's MHLW mark another pivotal milestone for Nanoscope. Sakigake recognition underscores the novelty of MCO-010 as a one-time, in-office intravitreal optogenetic therapy 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 for patients living with inherited retinal diseases."

Including these new designations in Japan, MCO-010 has also received 5 orphan designations from the EMA and orphan drug and fast track designations for retinitis pigmentosa from the FDA. Additionally, it has received orphan drug, fast track, and regenerative medicine advanced therapy (RMAT) designations for Stargardt disease (SD).

The company also announced that the EMA, FDA, and PDMA have approved the brand name MOGENRY for MCO-010.

Recently, Nanoscope received a new patent (US Patent No. 12459977) from the US Patent and Trademark Office (USPTO) for the company’s Multi-Characteristic Opsin (MCO) technology platform as a novel and innovative synthetic opsin for optogenetic modulation.²

Additionally, the company released positive long-term safety results from its EXTEND study, a 5-year follow-up of participants who received a single intravitreal injection of MCO-010 in a previously conducted phase 1/2a³ trial, as well as positive 3-year follow-up data from its REMAIN study, the long-term extension of the phase 2b/3 RESTORE trial (NCT04945772) evaluating MCO-010 in retinitis pigmentosa (RP).⁴

According to the company, results from EXTEND showed that a single intravitreal injection of MCO-010 is safe and well-tolerated over 5 years, with no serious adverse effects or new safety signals. The safety profile of MCO-010 remained consistent between the phase 1/2a trial and the long-term follow-up period.³

According to the company, results from the REMAIN study showed patients maintained an average best corrected visual acuity (BCVA) gain from baseline of approximately 0.3 LogMAR through week 152. Additionally, BCVA-Area Under the Curve (AUC) profiles across both the RESTORE and REMAIN trials show five times greater vision gains when compared to sham treatment.⁴

References:

1. Nanoscope Secures Japan MHLW Sakigake and Orphan Drug Designations Across Inherited Retinal Diseases - A First for a Retinal Gene Therapy. Published January 20, 2026. Accessed January 20, 2026. https://www.prnewswire.com/news-releases/nanoscope-secures-japan-mhlw-sakigake-and-orphan-drug-designations-across-inherited-retinal-diseases--a-first-for-a-retinal-gene-therapy-302664876.html

2. Harp MD. Nanoscope secures new US patent for Multi-Characteristic Opsin (MCO) technology platform. Published January 11, 2026. Accessed January 20, 2026. https://www.ophthalmologytimes.com/view/nanoscope-secures-new-us-patent-for-multi-characteristic-opsin-mco-technology-platform

3. Harp MD. Nanoscope Therapeutics releases positive long-term safety results from EXTEND study. Published November 5, 2025. Accessed January 20, 2026. https://www.ophthalmologytimes.com/view/nanoscope-therapeutics-releases-positive-long-term-safety-results-from-extend-study

4. Harp MD. Nanoscope Therapeutics releases positive 3-year follow-up data from REMAIN study. Published October 24, 2025. Accessed January 20, 2026. https://www.ophthalmologytimes.com/view/nanoscope-therapeutics-releases-positive-3-year-follow-up-data-from-remain-study