VX-152

VX-152 is a next-generation small molecule corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis. It functions as a CFTR corrector, aiming to restore proper folding and trafficking of the defective CFTR protein caused by mutations such as F508del. By improving the localization and function of CFTR at the cell surface, VX-152 increases chloride ion transport across epithelial cells. In clinical studies, it was evaluated in combination with tezacaftor (another corrector) and ivacaftor (a potentiator), targeting patients with at least one copy of the F508del mutation. The drug reached Phase 2 clinical trials but its development for cystic fibrosis has been discontinued[1][2][3][4][5][7].