CAS: 2095064-06-3

Posenacaftor (PTI-801) sodium, a CFTR modulator for CF research, corrects CFTR protein folding and trafficking.

Posenacaftor is a small molecule that belongs to the class of peptide inhibitors. It inhibits Protein interactions and is an activator of the ATP-binding cassette transporter A1 (ABCA1). Posenacaftor is used as a research tool to study the role of ABCA1 in lipid metabolism, as well as to study other proteins and their ligands. This drug has been shown to inhibit the activity of ion channels, which may have implications for treatment of epilepsy. Posenacaftor also binds to receptor sites on cells and blocks them, preventing calcium from entering into cells. This process leads to decreased inflammation and muscle contraction.