815592-21-3

VRT-325 is a novel small molecule discovered by screening a compound library designed to address the genetic defect in cystic fibrosis (CF) caused by the ΔF508 mutation in CFTR. VRT-325 belongs to a class of compounds that promote ΔF508-CFTR efflux from the endoplasmic reticulum and restores chloride transport levels in epithelial cells of CF-derived bronchi[1].

[1] Van Goor F, et al. Rescue of ?認508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules[J]. American Journal of Physiology-Lung Cellular and Molecular Physiology, 2006, 290(6): L1117-L1130.