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.
A phase 1 & 2 study to test the safety and tolerability of PTI-801 is underway.
This program is sponsored by Proteostasis Therapeutics. It is being conducted through the Cystic Fibrosis Foundation's Therapeutic Development Network.
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