Single intravitreal injection of MCO-010 produced sustained improvement in visually guided behavior in preclinical model of Stargardt disease
MCO-010-treatment showed significant increases in retinal physiologic response, with no notable change in retinal thickness, suggesting a potential disease-modifying effect
Findings reinforce MCO-010’s mutation-agnostic mechanism and intravitreal delivery advantage in a disease with no approved treatment
DALLAS, June 11, 2026 — Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced the publication in the journal, Bioengineering, results demonstrating that a single intravitreal injection of MCO-010, an investigational optogenetics therapy, significantly improved visual function and preserved retinal architecture in a mouse model of Stargardt disease.
Stargardt disease is the most common inherited juvenile macular degeneration, affecting approximately 1 in 8,000–10,000 individuals, with no FDA-approved treatment to date. Conventional gene replacement approaches need viable photoreceptors and are constrained by the ABCA4 gene’s large size, which exceeds standard AAV vector packaging capacity. MCO-010 sidesteps this limitation entirely by targeting surviving ON-bipolar cells downstream of degenerating photoreceptors.
In the study, Multi-Characteristic Opsin (MCO-010) treated Abca4⁻/⁻ mice navigated a radial water maze much faster than control mice with AAV2-vehicle treatment. Additionally, increases in B-wave amplitude indicating improvements in retinal function were observed, with no further decrease in retinal thickness, vs. a measurable retinal thinning in control mice. This effect is consistent with a potential disease-modifying mechanism. Immunohistochemistry confirmed robust, targeted MCO-010 expression in ON-bipolar cells of the inner retina at the end of the study.
“These results reinforce MCO-010’s potential not only to restore vision but to modify the course of retinal degeneration — an outcome that has not been demonstrated by any approved therapy,” said Samarendra Mohanty, PhD, Founder and President of Nanoscope Therapeutics. “The durability of response, combined with structural preservation of the retina, strengthens our confidence in MCO-010 as a platform therapy applicable across a broad spectrum of outer retinal degenerative diseases.”
The study findings are available online in Bioengineering ここ.
商業化に向けて前進
Nanoscope continues to build clinical, regulatory, and scaled-up manufacturing momentum in support of MOGENRY (MCO-010) for retinitis pigmentosa, including advancement of its rolling Biologics License Application in the U.S. The company has also established a growing global regulatory foundation for MOGENRY, with Orphan Drug, Fast Track, and RMAT designations in the U.S., and Sakigake and Orphan Drug designations in Japan, five EMA Orphan designations across inherited retinal dystrophies, and, most recently, an Orphan drug designation for IRDs in Saudi Arabia. These milestones reflect the company’s expanding international regulatory pathway as it advances commercialization planning across key global markets.
MCOプラットフォームについて
MCOは、網膜色素変性症（RP）、スターガルト病（SD）、地図状萎縮症（GA）などの光受容体変性症患者の視力回復を目的とした、疾患を問わない単回投与の硝子体内治療プラットフォームです。MCOは、高密度の双極網膜細胞を活性化して光感受性を持たせることで、光受容体死後に残存する視覚回路を活用します。MCO治療は、遺伝子検査、侵襲的な手術、または繰り返し投与を必要としないため、既存の網膜診療ワークフロー内で幅広い患者に適用可能です。.
ナノスコープ・セラピューティクスについて
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), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, 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 SD (NCT05417126）を開始し、 フェーズ3登録試験 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 to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. A Phase 2 program for MCO in GA is expected to start in 2026. Other IND-ready programs include Leber congenital amaurosis (LCA).
Contact: Nanoscope Therapeutics, Inc.
+1 (817) 857-1186
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