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.

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

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).

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.

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

Glucosamine (Chitosamine) is a natural product. Glucosamine is used as a dietary supplement. Glucosamine has pharmacological effects on cartilage and chondrocytes in osteoarthritis when used externally. Glucosamine regulates sugar metabolism. Glucosamine reduces the activity of glucoskinase in pancreatic islet homogenate.

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.

Corrector C4, a corrector commonly used to study cystic fibrosis mutants, works by alleviating the interaction between CFTR transmembrane domain mutants and protein homeostasis.

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.

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.

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.

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

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

5,7,4'-Trimethoxyflavone (4',5,7-Trimethoxyflavone) is isolated from Kaempferia parviflora (KP) which is a medicinal plant from Thailand.

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

Crinecerfont (SSR-125543) is a selective antagonist of corticotropin-releasing factor 1 receptor (CRF1) and can be used in studies about Classic congenital adrenal hyperplasia.

Kobusin, a bisepoxylignan compound isolated from Pnonobio biondii Pamp, demonstrates CFTR and CaCC chloride channel activation properties while exhibiting inhibitory effects on ANO1/CaCC channels.

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

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

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

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