Nanoscope Therapeutics Announces Completed Enrollment of its Phase 2b Clinical Trial in Blind Patients for Optogenetic Gene Therapy to Restore Vision


DALLAS, Feb. 7, 2022 /PRNewswire/ -- Nanoscope Therapeutics, a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced full enrollment of its Phase 2b clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP). Topline results are expected in Q1 of next year.

"Completing enrollment for this trial is another important milestone for Nansocope and millions suffering from retinal diseases for which there are no effective treatments," said Sulagna Bhattacharya, CEO of Nanoscope. "Positive results from this trial will bring us closer to meeting this significant unmet medical need."  

A Completed Phase 1/2a study of MCO-010 in 11 patients with RP demonstrated that the optogenetic therapy was well-tolerated, with improved quality of life consistent with significant functional vision improvement in advanced RP patients. 

The Phase 2b trial (NCT04945772) enrolled 27 participants with severe vision loss due to advanced RP in a randomized, double-masked, sham-controlled, multi-center trial. In this study, patients receive either an injection of MCO-010 or a sham injection to evaluate the efficacy and safety of MCO-010 therapy.

"RP is a progressive inherited condition caused by a spectrum of different gene mutations. This optogenetic therapy has the potential to impact patients with various genetic types of RP," said Byron Lam, MD, Neuro-Ophthalmology Professor at Bascom Palmer Eye Institute and leading expert on RP.

Nanoscope's optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells enabling vision in natural environments. The therapy is administered as a single eye injection for in-office delivery without the need for any other devices or interventions. Based on evidence from the Phase 1/2a study, MCO-010 is potentially applicable for vision restoration in retinal degenerative diseases irrespective of gene mutations. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA. A Phase-2 study in Stargardt macular degeneration is also being initiated.

About RP 
RP, linked to over 100 different gene mutations, encompasses a group of rare disorders where photoreceptors degrade, leading to blindness in approximately 2 million individuals.

About Nanoscope Therapeutics
Nanoscope Therapeutics is developing optogenetic therapies to restore sight in millions of blind individuals suffering from retinal diseases, for which no cure exists. The pipeline includes optogenetic therapies for RP, Stargardt, and dry-AMD.

Dan Eramian
Opus Biotech Communications


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SOURCE Nanoscope Therapeutics


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