|Description||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. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and cystic fibrosis (nmCF).|
|Appearance||White to off-white solid|
|Current Developer||PTC Therapeutics.|
BPO-27 racemate inhibited CFTR with IC50 ≈ 8 nM and had greatly improved in vitro metabolic stability and aqueous solubility compared to PPQ-102.
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....
iOWH-032 is a novel and potent CFTR inhibitor (IC50=1.01 uM) in T84 and CHO-CFTR cell based assays.
Ivacaftor benzenesulfonate is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.
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...
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...
VX-661 is a second F508del CFTR corrector and is believed to help CFTR protein reach the cell surface. Phase 2.
Ivacaftor hydrate is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.
Ivacaftor is a potentiator of CFTR targeting G551D-CFTR(EC50=100 nM)and F508del-CFTR(EC50=25 nM).
KM11060 is a mutated F508del cystic fibrosis transmembrane conductance regulator (CFTR) corrector. KM11060 is an analog of sildenafil, which restores a function...
(R)-BPO-27, the benzopyrimido-pyrrolo-oxazinedione, is a potent CFTR inhibitor (IC50= 4 nM) with low-nanomolar potency.
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...