Drug Development Pipeline
Restore CFTR Function
PTI-428 is a type of CFTR modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such as ivacaftor and lumacaftor, to work on. When combined with other therapies, PTI-428 may increase CFTR function across multiple gene mutations.
A phase 1 study to test the safety of PTI-428 has been completed.
This program is sponsored by Proteostasis Therapeutics. The early discovery phase was partially funded by the Cystic Fibrosis Foundation.
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