Posenacaftor

Posenacaftor (PTI-801) is a small molecule CFTR modulator developed by Proteostasis Therapeutics for the treatment of cystic fibrosis. It acts as a CFTR corrector, improving the folding and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to increase its function at the cell membrane. This mechanism addresses defects caused by mutations in the CFTR gene, particularly those leading to misfolded or dysfunctional protein that underlies cystic fibrosis pathology. Posenacaftor has been studied both alone and in combination with other modulators such as PTI-808 and PTI-428[1][4][5][6].

Other names

posenacaftorPTI-801 sodiumPTI801 sodiumPTI 801 sodium