Vanzacaftor CAS# 2374124-49-7

Vanzacaftor is a next-generation CFTR (cystic fibrosis transmembrane conductance regulator) modulator developed as a corrector for cystic fibrosis treatment. It is designed to enhance CFTR protein folding and trafficking, particularly in combination with other modulators like deutivacaftor and tezacaftor. In preclinical and clinical studies, vanzacaftor has demonstrated high potency in improving chloride transport in CFTR-mutant cells, particularly those with the common F508del mutation. Studies have reported significant increases in CFTR function, with EC50 values in the low nanomolar range, indicating strong bioactivity.