Posenacaftor (PTI-801) sodium, a cystic fibrosis transmembrane regulator (CFTR) modulator, corrects CFTR protein folding and trafficking. It is utilized in researching cystic fibrosis (CF)[1].

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene[1]. These mutations result in the production of a defective CFTR protein, impairing ion flow across cell membranes, particularly at the apical membrane of epithelial cells[1]. Posenacaftor serves as a CFTR corrector, a compound designed to repair and restore functional activity to the malfunctioning CFTR protein. Once corrected, CFTR can relocate to the cell surface, acting as a chloride channel to regulate fluid balance in the airways[2].