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 is underway to test the safety of PTI-428 in people with CF.
This program is sponsored by Proteostasis Therapeutics. The early discovery phase was partially funded by Cystic Fibrosis Foundation Therapeutics (CFFT).
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