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<\/p>\nFurther to its potential in Stargardt disease, if approved, the one-time gene therapy could become standard of care for retinitis pigmentosa, alongside other retinal degenerative diseases.<\/p>\n
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Nanoscope Therapeutics has announced that a first-of-a-kind study has demonstrated vision improvement in individuals with Stargardt disease.<\/p>\n
The genetic eye condition, also known\u00a0as juvenile macular dystrophy, is a major cause of vision loss in children. The Phase II STARLIGHT study<\/a> evaluated six participants given an intravitreally injected gene-agnostic MCO-010\u00a0(sonpiretigene isteparvovec) therapy.<\/p>\n MCO-010\u2019s mechanism of action enables highly dense bipolar retinal cells to become light sensitive, according to Nanoscope.<\/p>\n Notably, the gene therapy\u00a0provided an improvement in mean visual acuity of 12 ETDRS letters over 48 weeks in\u00a0patients with atrophy confined to the macula. No serious adverse events were reported and the treatment well tolerated.<\/p>\n Nanoscope also observed that some patients experienced vision improvement as early as four weeks post-treatment, which was maintained throughout the study duration.<\/p>\n Advancing gene therapy research in retinal diseases<\/p>\n this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes\u201d<\/p>\n Dr Byron Lam, Professor of Ophthalmology at Bascom Palmer Eye Institute, lead\u00a0author and principal investigator of the STARLIGHT study added: \u201cWhile this was an open-label study, this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes.\u201d<\/p>\n Nanoscope\u00a0plans to begin a Phase III trial in Stargardt disease by the end of 2025. Further to the Phase II STARLIGHT study, top-line results for the STARGAZE\u00a0Phase III study are expected in 2027.<\/p>\n This recent data from the STARLIGHT clinical trial was published in eClinical Medicine<\/a>.<\/p>\n