Properties
Description
VX-809, soluble in DMSO and ethanol and insoluble in water, blocks the activity of the cystic fibrosis transmembrane conductance regulator protein, which plays a crucial role in the movement of chloride ions across cell membranes. It targets F508del-CFTR (Fisher rat thyroid cells).
Molecular Formula
C24H18F2N2O5
Targets
F508del-CFTR (Fisher rat thyroid cells): 0.1 μM (EC50)
Solubility
DMSO: 88 mg/mL (194.51 mM); Water: Insoluble; Ethanol: 6 mg/mL (13.26 mM)
Identity
Confirmed by NMR/HPLC/MS.
Applications
VX-809 (Lumacaftor) is employed in studies as a CFTR corrector to investigate therapeutic strategies for cystic fibrosis by correcting the folding defect of mutant CFTR protein.