PTI-801 (alone and in combination)

PTI-801 is a new CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

PTI-801 is currently being studied both alone as well as in combination with two other CFTR modulators, PTI-428 and PTI-808.

Status

A phase 1 & 2 study to test the safety and tolerability of PTI-801 is underway.

Sponsor

This program is sponsored by Proteostasis Therapeutics. It is being conducted through the Therapeutics Development Network.