Phase 2 gene therapy trial for Stargardt disease underway – Healio


Press Release
Press Release
The first patient has been dosed in a phase 2 clinical trial investigating MCO-010 ambient light activatable optogenetic monotherapy for Stargardt disease, according to a press release from Nanoscope Therapeutics.
The STARLIGHT open-label trial will include approximately six patients set to receive 1.2E11 gc/eye of MCO-010, the same dose that was used in a phase 2b retinitis pigmentosa study. Safety and efficacy data for the first 6 months of the trial are expected in the first quarter of 2023.
“Existing experimental treatments for Stargardt primarily aim to slow progression of vision loss,” Aaron Osborne, Nanoscope chief medical officer and chief development officer, said in the release. “For people who already have advanced vision loss, an optogenetics-based approach, such as MCO-010, may have much greater potential to restore lost vision.”
The therapy uses vector and promoter technology to deliver the multi-characteristic opsin genes into retinal bipolar cells, reprograming healthy retinal cells to make them photosensitive and enabling vision in different color environments, the release said.
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