Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR), a protein kinase A (PKA)-activated epithelial anion channel involved in salt and fluid transport in multiple organs, including the lung. Most CF mutations either reduce the number of CFTR channels at the cell surface (e.g., synthesis or processing mutations) or impair channel function (e.g., gating or conductance mutations) or both. There are currently no approved therapies that target CFTR. Here we describe the in vitro pharmacology of VX-770, an orally bioavailable CFTR potentiator in clinical development for the treatment of CF. In recombinant cells VX-770 increased…

• CF
  Cystic Fibrosis Foundation
• CF
  Cystic Fibrosis Foundation Therapeutics
• VP
  Vertex Pharmaceuticals
• NI
  National Institutes of Health