CFTR

Elexacaftor (VX-445) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It promotes the processing and trafficking of CFTR, increases the amount of CFTR on the cell surface, and improves the processing and trafficking of Phe508del CFTR protein.

Tezacaftor (VX661) is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.

Ivacaftor (VX-770) (VX-770) is a potentiator of CFTR targeting G551D-CFTR (EC50: 100 nM) and F508del-CFTR (EC50: 25 nM) in Fisher rat thyroid cells, respectively.

Icenticaftor (QBW251) is an orally active potentiator of the CFTR channel.

Lumacaftor (VRT 826809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein. It enhances F508del-CFTR protein maturation in FRT cells (EC50: 100 nM).

CFTR(inh)-172 (CFTR Inhibitor-172) is a voltage-independent, selective CFTR inhibitor.

Glucosamine (Chitosamine) is an amino sugar and a prominent precursor in the biochemical synthesis of glycosylated proteins and lipids. Supplemental glucosamine may help to rebuild cartilage and treat arthritis.

CFTR corrector 12 is a CFTR corrector that rescues all mutant proteins except M760R ABCA3, and can be used to study cystic fibrosis.

Bamocaftor is a CFTR channel (DeltaF508-CFTR mutation) corrector for CF transmembrane conductance regulators designed to restore F508del-CFTR protein function. Combined use of tezacaftor and VX-561 for the treatment of cystic fibrosis patients with F508del MF.

PPQ-102 (CFTR Inhibitor), an effective CFTR inhibitor, can completely inhibit CFTR chloride current (IC50: 90 nM).

CFTR corrector 4 is a potent and orally available transmembrane conductance regulator (CFTR) for cystic fibrosis and is a potent (R,R) type active enantiomer. CFTR corrector 4 increases CFTR levels on the cell surface and is a potential compound for the study of cystic fibrosis.

Cavosonstat (N91115) is an orally active S-nitrosoglutathione reductase (GSNOR) inhibitor that promotes cystic fibrosis transmembrane conductance regulator (CFTR) maturation and plasma membrane stabilization. As a CFTR stabilizer, Cavosonstat can be used to study cystic fibrosis.

Glibenclamide (Glyburide) is an antidiabetic sulfonylurea derivative with actions similar to those of chlorpropamide.

(R)-BPO-27 is an orally active and potent ATP-competitive CFTR inhibitor (IC50: 4 nM) for the study of diarrhoea and polycystic kidney.

Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.

SRI-37240 is a potent inhibitor of premature termination codons (PTCs) with read-across activity that induces a prolonged pause at the termination codon and inhibits PTCs associated with cystic fibrosis.SRI-37240 can be used to study PTC-related diseases.

BPO-27 racemate (BPO-27 (racemate)) is an effective CFTR inhibitor with IC50 of 8 nM.

Nesolicaftor (PTI-428) specifically enhances cystic fibrosis transmembrane conductance regulator protein synthesis.

SRI-41315 is an eRF1 degrader that acts as a molecular glue at the center of ribosomal decoding.SRI-41315 induces an extended pause at the termination codon and inhibits cystic fibrosis-associated PTC in immortalized and primary human bronchial epithelial cells, restoring CFTR expression and function.SRI-41315 inhibits PTC by decreasing the abundance of the termination factor eRF1 NIOCH 14

GLPG1837 (ABBV-974) is an effective CFTR potentiator, with EC50s of 3 nM and 339 nM for F508del and G551D CFTR, respectively.

GlyH-101 is a cell-permeable glycinyl hydrazone compound that blocks CFTR with Ki of 1.4 uM.

CFTR corrector 2 (FDL169) is a novel and potent CFTR (Cystic fibrosis transmembrane conductance regulator) corrector for treating cystic fibrosis (CF) patients who carry the F508del mutation.

UCCF-853 is a small-molecule CFTR modulator that can be used to study cystic fibrosis.

IOWH-032 (IOWH032) , a synthetic CFTR inhibitor, has been investigated for the treatment of cholera, diarrhea, and secretory diarrhea.

KM11060 is a novel corrector of the F508del-CFTR trafficking defect, Correcting F508del-CFTR trafficking, increasing the amount of functional CFTR at the plasma membrane.

CFTR corrector 8 is a highly effective modulator of the cystic fibrosis transmembrane conductance regulator (CFTR). This compound is specifically designed for utilization in research related to cystic fibrosis, a genetic disorder primarily affecting the lungs and digestive system [1].

VRT-532 (CFpot-532) is an effective modulator of CFTR and is commonly used in studies of cystic fibrosis (CF) caused by CFTR defects. Modification of CFTR by this small molecule modulator can increase arylsulfatase B (ARSB), which is necessary to reduce the accumulation of sulphate glycosaminosaccharide (gag), thus reducing the accumulation of 4-chondroitin sulfate in cystic fibrosis.

DNDS is a channel blocker of voltage-dependent cystic fibrosis transmembrane conductance regulator (CFTR).

CFTR Corrector 9, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, is employed in the research of cystic fibrosis (CF) and related CFTR-associated disorders [1].

PG01 is a potent CFTR Cl-channel potentiator, effective against ΔF508 (Ka 0.3 μM), and also against E193K, G970R and G551D (CFTR mutants), with Kd values of 0.22 μM, 0.45 μM and 1.94 μM, respectively.PG01 increases ΔF508-CFTR Cl- currents upon addition of forskolin. F508-CFTR Cl-current upon addition of Forskolin, correcting the gating defect of CFTR mutants.

WAY-326766 increases ion transport through mutant CFTR and can be used to alter the lifespan of eukaryotes.WAY-326766 can be used to treat cystic fibrosis (CF).

(R)-Elexacaftor, an enantiomer of Elexacaftor, functions as a modulator of the cystic fibrosis transmembrane conductance regulator (CFTR). It exhibits an EC50 of 0.29 µM for the CFTR dF508 mutation [1].

CFTR Corrector 15 (Compound 4172) serves as a corrector for cystic fibrosis transmembrane conductance regulator (CFTR). When used in combination with VX-809, it addresses the folding defects of F508del-CFTR. CFTR Corrector 15 is also applicable in the research of cystic fibrosis.

CFTRcorrector 16 (Compound 39) is a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR), utilized in the research of cystic fibrosis disease.

CRF1 Receptor Antagonist-1 (Compound 2), a CRF1 receptor antagonist, is utilized in research pertaining to congenital adrenal hyperplasia (CAH) [1].

CFTRcorrector 17 (example 17) is a regulator of the cystic fibrosis transmembrane conductance regulator (CFTR). It is utilized in the study of diseases mediated by CFTR.

kCAL01 is a CAL inhibitor with a Ki value of 2.3 μM and holds potential for research in cystic fibrosis (CF).

CFTRactivator 2 (WAY-326769) acts as an activator for the mutant cystic fibrosis transmembrane conductance regulator protein (mutant-CFTR).