zuretinol acetate

ZA Low dose is the low-dose formulation of zuretinol acetate (also known as QLT091001, BBP-551, or EB-109), an orally administered synthetic retinoid replacement therapy. It is designed to treat inherited retinal diseases (IRDs) such as Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) caused by biallelic mutations in the RPE65 or LRAT genes. Zuretinol acetate is a synthetic prodrug of 9-cis-retinol, which is oxidized in vivo to 9-cis-retinal. This active form binds to opsin in photoreceptor cells to form isorhodopsin, a functional visual pigment analog that bypasses the genetic blocks in the visual cycle and restores phototransduction. Originally developed by QLT Inc., the program was subsequently developed by Novelion Therapeutics, BridgeBio Pharma, and Retinagenix Holdings, before being acquired by Eluminex Biosciences in January 2022. The compound has received FDA Rare Pediatric Disease and Fast Track designations.

Other names

9-cis-retinol9-cis-retinol acetate9-cis-retinyl acetateZA Low dosezuretinol