Rationalizing CF combos

A McGill University-led team has shown that Vertex Pharmaceuticals Inc.'s lumacaftor and other cystic fibrosis transmembrane conductance regulator corrector compounds target only one of two sequential steps required for proper folding of the mutated protein.¹ The findings suggest there is a clear rationale for developing combinations of correctors to treat patients with cystic fibrosis, three of which have been detailed at recent scientific meetings.

Cystic fibrosis is caused by mutations that reduce the function of cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel that helps keep the lung and intestinal epithelium hydrated and prevents mucous buildup that leads to airway obstruction and infection...