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.

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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. 

“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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