▎WuXiEdited by Kant Content Team

Nanoscope Therapeutics today announced that its optogenetics-based investigational gene therapy, MCO-010, met the primary and key secondary endpoints in the Phase 2b clinical trial RESTORE, significantly improving patients' vision.MCO-010 aims to treat permanent severe vision loss caused by advanced retinitis pigmentosa (RP) without considering the type of genetic mutation.Based on these positive results, the company plans to submit a Biologics License Application (BLA) to the U.S. FDA in the second half of this year. The press release also noted,This advancement represents a major breakthrough for optogenetics as a therapeutic modality.

After Week 52 of the study, the improvement in visual function persisted or increased, demonstrating the lasting effect of a single intravitreal injection of MCO-010. At Week 76, the improvement in BCVA in the high-dose treatment group was statistically significant compared to the control group (increase of 0.539 LogMAR; p=0.001). At Week 76, the improvement in BCVA in the low-dose treatment group was not statistically significant compared to the control group (increase of 0.374 LogMAR; p=0.065). These results are consistent with those observed in the earlier Phase 1/2a open-label study. The company plans to use the high dose of MCO-010 as the commercial dose.

Based on these results,Nanoscope Therapeutics Inc. plans to submit a Biologics License Application (BLA) to the U.S. FDA in the second half of 2024.

Existing gene therapies aim to treat patients carrying specific genetic mutations, but their application is limited in advanced diseases with degeneration of outer retinal cells. Optogenetic therapies that can be activated by ambient light target abundant inner retinal neurons and have the potential to permanently restore vision loss caused by advanced RP.MCO-010 expresses a broad-spectrum, fast, and highly sensitive opsin through transgenic means, localized in bipolar cells via the mGluR6 promoter-enhancer.The company's proprietary AAV2 vector allows for robust transduction of MCO-010 in bipolar cells following intravitreal injection. A Phase 1/2a clinical trial of MCO-010 in patients with advanced RP has demonstrated favorable safety and dose-dependent improvements in visual acuity.

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