Nanoscope Therapeutics’ MCO-010 holds potential to become new treatment option for retinitis: GlobalData

Nanoscope Therapeutics has recently submitted a rolling biologics license application (BLA) to the FDA for MCO-010 (sonpiretigene isteparvovec), a novel gene therapy candidate targeting retinitis pigmentosa (RP). Currently, the RP treatment landscape includes only one FDA-approved therapy, Luxturna (voretigene neparvovec), which is indicated for a small subset of patients with biallelic RPE65 mutations. In contrast, MCO-010 is poised to expand therapeutic options with a distinct mechanism of action and potentially broader applicability, says GlobalData.

MCO-010 is based on Nanoscope’s proprietary Multi-Characteristic Opsin (MCO) platform, utilizing a broadband light-sensitive “white opsin” delivered via an engineered adeno-associated virus (AAV) vector. The gene construct incorporates a fusion of three opsins—ChR2 (blue light), C1V1 (green light), and ReaChR (red light)—each with spectrally distinct activation peaks. This design enables responsiveness to ambient white light, potentially restoring visual function under natural lighting conditions.

MCO-010 works by transducing retinal cells with the white opsin gene, allowing light-evoked activation even in degenerated photoreceptors, eliciting robust photocurrents at physiologic light intensities, supporting functional vision restoration while minimizing light-induced retinal stress. The therapy is administered via a single intravitreal injection and is currently undergoing long-term evaluation in the Phase IV REMAIN study, following completion of the Phase IIb/III RESTORE trial. These studies aim to assess long-term efficacy, pharmacokinetics, pharmacodynamics, and safety. The REMAIN trial comprises three cohorts (n=27): Cohort 1 (n=9) received the high dose (1.2E11 gc/eye), Cohort 2 (n=9) received a lower dose (0.0E11 gc/eye), and Cohort 3 (n=9) serves as the sham control group.

The latest interim results of the long-term follow-up study showed that both low- and high-dose cohorts of MCO-010 demonstrated significant and sustained improvements in best-corrected visual acuity–area under the curve (BCVA-AUC) profiles over a follow-up period approaching two and a half years. A single administration of MCO-010 at either dose level was associated with meaningful and durable gains in visual acuity among patients with permanent vision loss due to advanced retinitis pigmentosa and associated photoreceptor degeneration. Furthermore, MCO-010 is well-tolerated across both dose levels, with no serious adverse events reported throughout the study duration.

Sara Reci, MSc, Managing Pharma Analyst at GlobalData, comments, “This follow-up is critical for validating the durability of MCO-010’s effects and further supporting regulatory review, thereby substantiating MCO-010’s long-term use in patients with RP.”

Key opinion leaders (KOLs) interviewed by GlobalData acknowledged that Nanoscope has made notable progress in advancing MCO-010, and have expressed their interest in the therapy’s potential, but highlighted common concern across optogenetic approaches, specifically relating to the uncertainty surrounding the level of visual resolution these therapies can ultimately provide to patients.

Reci concludes, “With its innovative optogenetic mechanism and potential applicability across a genetically diverse RP population, MCO-010 represents a significant advancement in the field of retinal gene therapy—particularly for patients with no currently approved treatment options. Should the BLA submission be approved, MCO-010 would become the first intravitreal optogenetic therapy on the market. Its potential to restore meaningful visual function using ambient light could redefine therapeutic expectations for late-stage RP and establish a new paradigm for vision restoration in degenerative retinal diseases.”