FDL-169 is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector being developed by Flatley Discovery Lab for treating cystic fibrosis (CF) patients who carry the F508del mutation, the most common mutation in this disease.
Ivacaftor (also known as VX-770; trade name: KALYDECO and Symdeko) is a potent and orally active potentiator of CFTR (cystic fibrosis transmembrane conductance regulator) targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM in fisher rat thyroid cells, respectively.
PPQ-102 (also known as CFTR Inhibitor) is a potent inhibitor of CFTR (cystic fibrosis transmembrane conductance regulator) discovered from screening of approximately 110000 small synthetic and natural compounds for inhibition of halide influx in CFTR-expressing epithelial cells.
KM11060 is a novel mutated corrector of the F508del-CFTR (cystic fibrosis transmembrane conductance regulator) trafficking defect.
Elexacaftor (VX-445, WHO-11180; WHO11180; VX445; Kaftrio; Trikafta) is one of three components in the fixed-dose combination medication (Elexacaftor/tezacaftor/ivacaftor, trade name: Trikafta and Kaftrio) used in patients who have cystic fibrosis with a F508del mutation.
PTI-428 is a novel, potent CFTR amplifier being developed by Proteostasis Therapeutics, Inc.
Ivacaftor (also known as VX-770; trade name: KALYDECO and Symdeko) is a potent and orally bioactive potentiator of CFTR (cystic fibrosis transmembrane conductance regulator) targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM in fisher rat thyroid cells, respectively.
GLPG1837 (also known as ABBV-974) is a novel, potent and reversible CFTR (cystic fibrosis transmembrane conductance regulator) potentiator with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively.
GlyH-101 is a novel and potent CFTR inhibitor (cystic fibrosis transmembrane conductance regulator) with Ki of 1.4 uM.