PTI-130

PTI-130 is an investigational small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) amplifier originally developed by Proteostasis Therapeutics for the treatment of cystic fibrosis. As a CFTR amplifier, PTI-130 is designed to increase the amount of CFTR protein by boosting its expression, thereby raising the cellular CFTR protein load available for downstream correctors and potentiators to act on, and enhancing CFTR-mediated ion transport in airway epithelial cells.[1][3][9][17] Preclinical studies have shown that PTI-130 has favorable pharmacologic properties suitable for oral administration and a promising safety and tolerability profile in short-term animal toxicology studies, but the program has remained in preclinical or early development without clear progression into later-stage clinical trials.[1][5][15]