Nanoscope Therapeutics has dosed the first participant in a Phase II trial of its MCO-010 clinical stage gene therapy to treat Stargardt disease.
The Phase II STARLIGHT open-label trial has been designed to evaluate safety and efficacy of a single intravitreal injection of MCO-010 Optogenetic Therapy in Stargardt disease patients.
The MCO-010 gene therapy reprogrammes healthy retinal cells for making them photosensitive.
Using proprietary vector and promoter technology, the therapy delivers the Multi-Characteristic Opsins (MCO) genes into retinal bipolar cells to enable the vision in different colour environments.
It has received orphan drug designations from the US Food and Drug Administration (FDA) for Retinitis Pigmentosa (RP) and Stargardt diseases.
About six participants will be enrolled in the Phase II trial, which currently has active clinical sites in Texas and Florida, US.
In the trial, the participants will receive the same MCO-010 dose of 1.2E11gc/eye which was also used in the Phase IIb RP study.
The data from the six-month study is expected in the first quarter of next year.
Nanoscope Therapeutics advisor Stephen Tsang said: “MCO-010 offers new hope for individuals with Stargardt macular degeneration.
“Stargardt patients with severe degeneration typically lack sufficient light sensing photoreceptor neurons needed to qualify for experimental ABCA4 gene specific therapies.
“Optogenetic therapy may be able to treat a wider group of patients with Juvenile macular degeneration regardless of the status of their light sensing photoreceptor neurons.”
At present, the company’s gene therapy is also being evaluated in double-masked, multicentre, sham-controlled, randomised Phase IIb clinical trials in the US to treat RP.
Top line data from the study is expected in the first quarter of next year.
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