04/14/2026
A novel experimental therapy developed from foundational research at the UW Medicine Department of Ophthalmology has reached an important clinical milestone, with results from a first-in-human study published on April 14 in Nature Medicine. The paper reports early clinical findings for KIO-301, a light-activated small molecule designed to ultimately restore some vision in people with advanced retinitis pigmentosa. This rare inherited disease causes progressive vision loss.
Russell N. Van Gelder, MD, PhD, Boyd K. Bucey Professor and Chair of the Department of Ophthalmology in the University of Washington School of Medicine - WWAMI, who co-authored the paper, helped develop the underlying science behind this approach. The technology is based on a class of compounds known as molecular photoswitches, which can confer light sensitivity to surviving retinal cells after photoreceptors have been lost. Photoswitches are effectively nanometer-scale light switches that can turn drug activity on and off with light. KIO-301 targets voltage-gated potassium channels, which control neuronal firing.
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