km-11060

Description: KM11060 is a novel mutated corrector of the F508del-CFTR (cystic fibrosis transmembrane conductance regulator) trafficking defect. It corrects F508del-CFTR trafficking, and increases the amount of functional CFTR at the plasma membrane (ca.75%) and inhibits PDE5 activity. Small-molecule correctors such as KM11060 may serve as useful pharmacological tools in studies of the F508del-CFTR processing defect and in the development of cystic fibrosis therapeutics. KM11060 partially corrects F508del-CFTR processing and increases surface expression to 75% of that observed in cells incubated at low temperature. Up to 50% of the F508del-CFTR in cells treated with KM11060 was complex-glycosylated, indicating passage through the Golgi. KM11060 as a promising compound for further development of CF therapeutics.

References: Mol Pharmacol. 2008 Feb; 73(2):478-89.