REV-0100

REV-0100 is an intravitreal, repurposed small-molecule drug in early clinical development for Stargardt disease and potentially dry age-related macular degeneration. It is a generally recognized as safe (GRAS) compound discovered at Weill Cornell Medicine and licensed to reVision Therapeutics, designed to bind and clear toxic lipid bisretinoids (lipofuscin) that accumulate in retinal pigment epithelial cells in ABCA4-associated Stargardt disease, thereby reducing retinal cell death and slowing or preventing central vision loss.[2][3][4][5] REV-0100 has received Orphan Drug and Rare Pediatric Disease designations from the US FDA for Stargardt disease and is being co-developed through collaborations including reVision Therapeutics and Aequus Pharmaceuticals, with a lead indication in Stargardt disease where there are currently no approved treatments.[1][2][3][4][5][7][11]