Nanoscope Reveals MCO-010 Treatment Halts Neurodegeneration

DALLAS, Oct. 21, 2024 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology firm focusing on gene therapies for retinal degenerative diseases, has revealed a significant advancement in treating these conditions. The company's latest findings, published in Translational Vision Science & Technology (TVST), highlight the efficacy of their optogenetic treatment, MCO-010, in halting further retinal degeneration in an animal model of retinitis pigmentosa (RP).

MCO-010, also known as sonpiretigene isteparvovec, is an optogenetic therapy administered via intravitreal injection. The study demonstrated that approximately 80% of bipolar cells in the retina were successfully transduced with MCO-010, without any observed changes in retinal thickness, contrasting the control group. This suggests that the therapy can maintain retinal structure and prevent the progression of degeneration.

The publication provides compelling evidence that MCO-010 can stop further disorganization and loss of retinal cell layers in mice experiencing photoreceptor degeneration. This is a crucial finding as it points to the potential of MCO-010 to not only restore vision but also to modify the disease course by targeting the inner retina's bipolar cells.

Dr. Vinit Mahajan, a Professor and Vice Chair for Research at Stanford University's Department of Ophthalmology, emphasized the unique properties of MCO-010. He highlighted that it is the only broadband, fast, and highly sensitive opsin currently in clinical trials. Dr. Mahajan also noted the significant improvements in vision observed in advanced RP patients following MCO-010 treatment, assessed through behavioral and electrophysiological tests. This dual capability of restoring vision and halting disease progression marks a vital therapeutic advantage in optogenetic therapies.

Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope, added that the disease-modifying effects of MCO-010 have been seen not just in RP but also in models of Stargardt disease and geographic atrophy in nonhuman primates. Preliminary evidence from the RP clinical trial indicates a stabilization of retinal structure, prompting plans for a long-term evaluation of this anatomical biomarker.

Nanoscope Therapeutics is at the forefront of developing gene-agnostic, sight-restoring optogenetic therapies for millions suffering from retinal degenerative diseases. These diseases currently have no cure, but Nanoscope's innovative approach offers hope. The company recently completed the RESTORE Phase 2b clinical trial for RP (NCT04945772) with positive outcomes and plans to submit a Biologics License Application (BLA) for MCO-010 to the FDA in the first quarter of 2025. This therapy has already received FDA Fast Track and orphan drug designations for both RP and Stargardt disease.

Additionally, Nanoscope has concluded the Phase 2 STARLIGHT trial of MCO-010 in Stargardt patients (NCT05417126) and is preparing to begin a Phase 3 registrational trial in early 2025. Their preclinical pipeline also includes non-viral, laser-delivered MCO-020 gene therapy for geographic atrophy.

Nanoscope Therapeutics continues to advance its mission of developing groundbreaking therapies that could change the lives of those afflicted by retinal degenerative diseases. The promising results from their latest research highlight the potential of MCO-010 to not only improve vision but also to alter the disease's trajectory, offering a beacon of hope for patients worldwide.