Nanoscope Therapeutics has concluded subject enrolment in a Phase IIb trial of its optogenetic gene therapy, MCO-010, to restore vision in patients with retinitis pigmentosa (RP).
The company anticipates topline data from the trial in the first quarter of next year. A total of 27 subjects with severe vision loss due to advanced RP were enrolled into the trial.
The randomised, sham-controlled, double-masked, multicentre trial will analyse the safety and efficacy of the MCO-010 therapy in RP patients.
Participants will be administered either an MCO-010 injection or a sham injection.
Nanoscope Therapeutics CEO Sulagna Bhattacharya said: “Completing enrollment for this trial is another important milestone for Nansocope and millions suffering from retinal diseases for which there are no effective treatments.
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“Positive results from this trial will bring us closer to meeting this significant unmet medical need.”
The concluded Phase I/IIa trial that enrolled 11 RP patients showed MCO-010 was well-tolerated, enhanced quality of life consistent with substantial improvement in functional vision in individuals with advanced disease.
As per this trial data, MCO-010 could be applicable for restoring vision in retinal degenerative diseases regardless of gene mutations.
The optogenetic monotherapy is an ambient-light activatable Multi-Characteristic Opsin (MCO).
It leverages an adeno-associated virus type 2 (AAV2) vector to offer MCO genes into retinal cells facilitating vision in natural environments.
The treatment is given as a single eye injection for in-office delivery without requiring any other devices or interventions.
The US Food and Drug Administration granted orphan drug designations for MCO-010 to treat RP and Stargardt disease.
Nanoscope also commenced a Phase II trial of the therapy in Stargardt macular degeneration.
Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.
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