Nanoscope Publishes Breakthrough Showing MCO-010 Treatment Arrests Neurodegeneration

Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases today announced a breakthrough discovery in Translational Vision Science & Technology (TVST, an ARVO journal) demonstrating that MCO-010 optogenetic treatment of the inner retina arrests further retinal degeneration in an animal model of retinitis pigmentosa (RP), as assessed by optical coherence tomography and immunohistochemical analysis.

Following intravitreal MCO-010 treatment (sonpiretigene isteparvovec), approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed, unlike the control group. The results described in the publication provide evidence that the expression of MCO-010 in the retinas of mice experiencing photoreceptor degeneration halts further loss and prevents further disorganization of the retinal cell layers.

“MCO-010 is the only broadband, fast, and highly sensitive opsin currently in clinical trials and has demonstrated significant improvement in vision in advanced RP patients. In addition to restoring vision by MCO-010 intravitreal injection, measured by behavioral and electrophysiological assays, this disease-modifying aspect of MCO-010 provides a key therapeutic advantage in optogenetic therapies by targeting bipolar cells in the inner retina,” said Dr. Vinit Mahajan, Professor and Vice Chair for Research, Department of Ophthalmology at Stanford University.

“This disease-modifying aspect of MCO-010 has also been observed in an animal model of Stargardt disease as well as the nonhuman primate model for geographic atrophy,” said Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope. “We have preliminary evidence of stabilization of retina structure in the RP clinical trial, and we plan to conduct a long-term evaluation of this anatomical biomarker.”

About Nanoscope Therapeutics, Inc.

Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate BLA submission for MCO-010 to treat RP in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

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