Olacaftor (VX-440, VX440) is a next-generation CFTR corrector, shows the potential to enhance the amount of CFTR protein at the cell’s surface and for treatment of cystic fibrosis. Fibrosis Phase 2 Clinical
Ivacaftor hydrate is an orally bioavailable CFTR potentiator, used for cystic fibrosis treatment.
Ivacaftor benzenesulfonate is an orally bioavailable CFTR potentiator, used for cystic fibrosis treatment.
Aloisine A (RP107) is a a potent cyclin-dependent kinase (CDK) inhibitor with IC50s of 0.15 μM, 0.12 μM, 0.4 μM, 0.16 μM for CDK1/cyclin B, CDK2/cyclin A, CDK2/cyclin E, CDK5/p35, respectively. Aloisine A ininhibits GSK-3α (IC50=0.5 µM) and GSK-3β (IC50=1.5 µM). Aloisine A stimulates wild-type CFTR and mutated CFTR, with submicromolar affinity by a cAMP-independent mechanism. Aloisine A has the potential for CFTR-related diseases, including cystic fibrosis research[1][2].
CFTR corrector 9 (compound 42) is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. CFTR corrector 9 can be used for researching cystic fibrosis (CF) and other CFTR associated disorders[1].
Vanzacaftor is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR) for treating cystic fibrosis.
(Rac)-Tezacaftor ((Rac)-VX-661) is a racemate of Tezacaftor (HY-15448). Tezacaftor is a F508del CFTR corrector. (Rac)-Tezacaftor can be used for the research of cystic fibrosis[1].
Crinecerfont (SSR-125543) hydrochloride is a potent, orally active, non-peptide CRF1 receptor antagonist. Crinecerfont can be used for Classic congenital adrenal hyperplasia (CAH) research[1].
Bamocaftor (VX-659) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore F508del-CFTR protein function. Bamocaftor can be used combine with Tezacaftor and Ivacaftor in cystic fibrosis research[1].
CFTR activator 1 is a potent and selective CFTR activator, with an EC50 of 23 nM. CFTR activator 1 can be used to ameliorate dry eye disease[1].
Icenticaftor (QBW251) is an orally active CFTR channel potentiator, with EC50s of 79 nM and 497 nM for F508del and G551D CFTR, respectively. Icenticaftor can be used for chronic obstructive pulmonary disease (COPD) and cystic fibrosis research[1].
(R)-Elexacaftor is an enantiomer of Elexacaftor (HY-111772). (R)-Elexacaftor is the Compound 37 from patent WO2018107100A1. (R)-Elexacaftor is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR), the EC50 for CFTR dF508 is 0.29 uM[1].
Lumacaftor (VX-809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein.
Galicaftor (ABBV-2222; GLPG-2222) is a potent and orally active cystic fibrosis transmembrane conductance regulator (CFTR) corrector. Galicaftor can be used for cystic fibrosis research[1].
Chromanol 293B is a selective blocker of the slow delayed rectifier K+ current (IKs) with IC50 of 1-10 μM and a weak inhibitor of KATP channel. Chromanol 293B also blocks the CFTR chloride current with an IC50 of 19 μM[1].
CFTR corrector 1 (compound 1) is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR). CFTR corrector 1 (compound 1) facilitates the processing and trafficking of CFTR to increase the amount of CFTR at the cell surface[1].
Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.
Astressin 2B is a potent and selective corticotropin-releasing factor receptor 2 (CRF2) antagonist, with the IC50 values of 1.3 nM and > 500 nM for CRF2 and CRF1, respectively. Astressin 2B antagonizes CRF2-mediated inhibition of gastric emptying[1][2][3].
Zatonacaftor is a modulator of cystic fibrosis transmembrane regulator (CFTR) protein. Zatonacaftor can be used for research of cystic fibrosis[1][2].
PPQ-102 is a potent CFTR inhibitor which can completely inhibited CFTR chloride current with IC50 of ~90 nM. IC50 value: 90 nM [1]Target: CFTRin vitro: The most potent compound, 7,9-dimethyl-11-phenyl-6-(5-methylfuran-2-yl)-5,6-dihydro-pyrimido[4',5'-3,4]pyrrolo[1,2-a]quinoxaline-8,10-(7H,9H)-dione, PPQ-102, completely inhibited CFTR chloride current with IC(50) approximately 90 nM. The PPQs, unlike prior CFTR inhibitors, are uncharged at physiological pH, and therefore not subject to membrane potential-dependent cellular partitioning or block efficiency. Patch-clamp analysis confirmed voltage-independent CFTR inhibition by PPQ-102 and showed stabilization of the channel closed state [1]. The three gpSlc26 anion transporters exhibited distinct pharmacological profiles of (36)Cl(-) influx, including partial sensitivity to CFTR inhibitors Inh-172 and GlyH101, but only Slc26a11 was inhibited by PPQ-102 [2]. Airway epithelial NCI-H292 cells and primary cultures of noncystic fibrosis human airway epithelial cells were treated with cystic fibrosis transmembrane conductance regulator (CFTR) inhibitors (CFTR-inh(172) or PPQ-102) or transfected with a CFTR small interfering (si)RNA with or without a selective epidermal growth factor receptor tyrosine kinase inhibitor [3].in vivo: PPQ-102 prevented cyst expansion and reduced the size of preformed cysts in a neonatal kidney organ culture model of polycystic kidney disease. PPQ-102 is the most potent CFTR inhibitor identified to date [1].
Glibenclamide (Glyburide) potassium is a potassium salt of Glibenclamide (HY-15206). Glibenclamid potassium exists in anhydrous and hydrate forms, with higher solubility compared to pure Glibenclamide[1].
CRF1 receptor antagonist-1 (Compound 2) is a CRF1 receptor antagonist. CRF1 receptor antagonist-1 can be used for research of congenital adrenal hyperplasia (CAH)[1].
SRI-37240 is a potent premature termination codons (PTCs) inhibitor. SRI-37240 suppresses CFTR nonsense mutations. SRI-37240 alters cellular translation termination at PTCs in HEK293T cells. SRI-37240 can also restore CFTR function in primary bronchial epithelial cells when combination with G418[1].
CFTR(inh)-172 is a potent and selective blocker of the CFTR chloride channel; reversibly inhibited CFTR short-circuit current in less than 2 minutes with a Ki of 300 nM.