PTI-801 + PTI-808

PTI-801 + PTI-808 is a **combination of two small molecule investigational drugs** developed as cystic fibrosis transmembrane conductance regulator (CFTR) modulators for the treatment of cystic fibrosis (CF)[1][2][3][4][5][6][7][8][9]. PTI-801 is a third-generation CFTR corrector, which helps repair the folding and function of the defective CFTR protein in patients with CF, especially those homozygous for the F508del mutation[9]. PTI-808 is a novel CFTR potentiator, which increases the channel activity of CFTR on the cell surface, improving chloride transport[9]. When used together, PTI-801 and PTI-808 address different defects in CFTR, with the goal of restoring adequate protein expression and function in patients. This combination, sometimes tested alongside PTI-428 (a CFTR amplifier), has been studied in early-phase trials and showed improvements in pharmacokinetics and relevant clinical endpoints in cystic fibrosis[2][5][9].

Other names

PTI-801PTI801PTI 801PTI-808PTI808PTI 808PTI-801 + PTI-808Dirocaftor + Posenacaftor