Identifying the Molecular Target Sites for CFTR Potentiators GLPG1837 and VX-770

Identification of the binding sites for small molecules that alleviate gating defects in CFTR would assist rational drug design for the treatment of cystic fibrosis. Yeh et al. identify two potential binding sites for prototypical CFTR potentiators at the interface of CFTR’s two transmembrane domains.