CFTRcorrector 4 (Compound 13), an active (R,R)-form enantiomer, is a highly potent and orally activecystic fibrosis transmembrane conductance regulator (CFTR)corrector.CFTRcorrector 4 can increaseCFTRlevels at the cell surface and have the potential for treatment of cystic fibrosis^[1].

EC50:130 nM (CFTR)^[1]

CFTR corrector 4 possesses high potency and efficacy with a EC₅₀of 0.028 μM in this HBE-TECC assay that assess the CFTR function, this lead resides in undesirable drug-like space with high molecular weight, and high HPLC Log D, indicating high lipophilicity. HBE-TECC: (Human Bronchial Epithelial-Trans-Epithelial Current Clamp) assay^[1].
CFTR corrector 4 is tested its potency and efficacy in human bronchial epithelial (HBE) cells with a EC₅₀value 130 nM in the cell surface expression (CSE-HRP) assay^[1].

539.52

Solid

C₂₉H₂₇F₂NO₇

1918142-34-3

Room temperature in continental US; may vary elsewhere.