Dual Stem Cell Scaffold Restores Vision in First-in-Human Trial for Aniridia-Related Keratopathy

A non-randomized clinical trial conducted at Moorfields Eye Hospital and University College London evaluated a collagen scaffold engineered to deliver two types of stem cells—limbal epithelial and stromal—directly to the corneas of patients with aniridia-related keratopathy (ARK). ARK, a progressive form of aniridia caused by mutations in the PAX6 gene, affects roughly one in 40,000 to one in 100,000 people and leads to severe corneal opacity. Nine participants each received the treatment in one eye, while the fellow eye served as an untreated control. The study’s findings were published in JAMA Ophthalmology on July 23, 2026.

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• Ocular Surface Score (OSS): Baseline average was 9.4 in treated eyes and 8.4 in untreated eyes (scale 0-15; 11-15 = severe). After three months, treated eyes improved to an average 5.9, and at twelve months to 6.7. Untreated eyes changed little, reaching 8.0 at twelve months.

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• Visual Acuity (ETDRS): Treated eyes gained an average of 24 letters, equivalent to nearly five lines on a standard vision chart.

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• Control Comparison: The untreated eyes showed no comparable improvement in either OSS or ETDRS scores over the same periods.

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These outcomes indicate measurable restoration of corneal surface health and visual function in a condition previously lacking effective therapy.

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• “This is the first-ever optical use of a collagen scaffold containing two types of stem cells and has proved effective for a previously untreatable progressive condition leading to sight loss. We are now looking to take this forward to a larger clinical trial, with a view to this being accepted as an NHS treatment.” — UCL Institute, of Ophthalmology

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The research team plans a larger, multi-center trial to confirm safety and efficacy across a broader patient population. Funding for the next phase is expected to come from the Medical Research Council, Moorfields Eye Charity, and the NIHR Biomedical Research Programme, with the aim of establishing the treatment as a standard NHS option for ARK.

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