zuretinol

Zuretinol (also known as zuretinol acetate, QLT091001, and other synonyms) is a synthetic retinoid developed as an oral replacement therapy for inherited retinal diseases caused by gene mutations that disrupt the availability of 11-cis-retinal. It acts as a functional substitute for missing or deficient 11-cis-retinal in the visual cycle, aiming to restore visual function in conditions such as Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP), particularly those associated with RPE65 or LRAT gene mutations. Mechanistically, zuretinol is a derivative of vitamin A (retinoid) that can bind to both retinoic acid receptor (RAR) and retinoid X receptor (RXR), thereby regulating gene transcription involved in vision and cellular differentiation. The drug has received orphan drug designation for LCA and RP from regulatory agencies[3][4][5].

Other names

zuretinol acetatesynthetic retinaldehydesynthetic retinoid