EYE-003 is an orally active RPE65 inhibitor with an IC50 value of 102 nM. It regulates the visual cycle in mice by decreasing the synthesis of 11-cis-Retinal (11cRAL) and increasing all-trans-retinyl esters (atREs). EYE-003 also inhibits dark-adapted electroretinogram (ERG) b-wave amplitudes and provides protection against retinal degeneration in Abca4^/^Rdh8^/^ mice by dose-dependently reducing retinal autofluorescent spots and preserving outer nuclear layer (ONL) thickness. EYE-003 is applicable for researching retinal disorders related to the visual cycle, such as Stargardt disease type 1 (STGD1).
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