EYE-003

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) .