|Description||Ivacaftor benzenesulfonate is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.|
VX-661 is a second F508del CFTR corrector and is believed to help CFTR protein reach the cell surface. Phase 2.
VX-809 is the second investigational oral candidate compound for the treatment of cystic fibrosis (CF). VX-809 may act to restore the function of the cystic fib...
PG 01 is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. PG 01 is effective on G970R although with a significant decrease in potency r...
Ivacaftor hydrate is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.
Ivacaftor benzenesulfonate is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.
BPO-27 racemate inhibited CFTR with IC50 ≈ 8 nM and had greatly improved in vitro metabolic stability and aqueous solubility compared to PPQ-102.
iOWH-032 is a novel and potent CFTR inhibitor (IC50=1.01 uM) in T84 and CHO-CFTR cell based assays.
(R)-BPO-27, the benzopyrimido-pyrrolo-oxazinedione, is a potent CFTR inhibitor (IC50= 4 nM) with low-nanomolar potency.
N-2-Naphthalenyl-glycine 2-[(3,5-dibromo-2,4-dihydroxyphenyl)methylene]hydrazide is a glycine hydrazide that has recently been shown to block CFTR channels. How...
Ivacaftor is a potentiator of CFTR targeting G551D-CFTR(EC50=100 nM)and F508del-CFTR(EC50=25 nM).
CoPo 22 is a corrector and potentiator for ∆F508-cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel function in cystic fibrosis.
KM11060 is a mutated F508del cystic fibrosis transmembrane conductance regulator (CFTR) corrector. KM11060 is an analog of sildenafil, which restores a function...
CFTRinh-172 is a voltage-independent, selective CFTR inhibitor with Ki of 300 nM, showing no effects on MDR1, ATP-sensitive K+ channels, or a series of other tr...
Ataluren is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation....