CFTR mutations are responsible for cystic fibrosis. The most common mutation is ΔF508, but many others exist. Drugs which can correct channel function of a broad range of CFTR mutants are most desirable for clinical development. PG-01 is a potentiator of ΔF508 (Ka 70 nM) as well as mutants G551D and G1349D (Ka 1100 and 40 nM, respectively). Its broad spectrum may make it more desirable than more mutant-specific CFTR correctors.
PG-01 is a potentiator of ΔF508 (Ka 70 nM) as well as mutants G551D and G1349D (Ka 1100 and 40 nM, respectively).
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