cystic fibrosis

Elexacaftor (VX-445) is a modulator of cystic fibrosis transmembrane conductance regulator (CFTR) and it also facilitates the processing and trafficking of CFTR to increase the amount of CFTR at the cell surface.

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

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).

Ibuprofen (Advil) is a propionic acid derivate and nonsteroidal anti-inflammatory drug (NSAID) with anti-inflammatory, analgesic, and antipyretic effects. Ibuprofen inhibits the activity of cyclo-oxygenase I and II, resulting in a decreased formation of precursors of prostaglandins and thromboxanes.

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.

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.

Tauroursodeoxycholate (UR 906), also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

IFB-088 acetate, a benzyl guanidine derivative, is used to treat diseases and cancers associated with the PPP1R15A pathway and associated with protein misfolding stress, Examples include tau disease, synaptic riboprotein disease, polyglutamine and polyalanine diseases, leukodystrophy, cystic fibrosis, multiple sclerosis, lysosome storage disorders, amyloidosis diseases, inflammation, metabolic disorders, cardiovascular diseases, osteoporosis, neurological trauma, and more.

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

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).

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.

CFTR corrector 12 is a CFTR corrector that rescues all mutant proteins except M760R ABCA3, and 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.

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.

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 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].

SSR69071 is a potent, orally active, and selective inhibitor of neutrophil elastase, displaying higher affinity for human elastase (Ki = 0.0168 nM) compared to rat (Ki = 3 nM), mouse (Ki = 1.8 nM), and rabbit (Ki = 58 nM) elastases [2]. It reduces myocardial infarct size following ischemia-reperfusion injury [1] and has potential for treating chronic obstructive pulmonary diseases, asthma, emphysema, cystic fibrosis, and various inflammatory diseases.

JNJ-49095397 (RV568) is a selective p38 MAPK-α and p38 MAPK--γ kinase inhibitor with anti-inflammatory activity and potential antiviral activity for chronic obstructive pulmonary disease (COPD), cystic fibrosis, and respiratory syncytial virus infection.

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

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

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

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

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

Ibuprofen Lysine (Neoprofen) is a non-steroidal anti-inflammatory drug.

Talniflumate (Somalgen) is a calcium-activated chloride channel (hCLCA1/mCLCA3) blocker that reduces mucin synthesis and releases in animal models and cell culture. It possesses anti-inflammatory actions via inhibition of Cox (cyclooxygenase) and inhibits Cl-/HCO3- exchange activity. It also increases survival in a cystic fibrosis mouse model of the distal intestinal obstructive syndrome.

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.

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

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

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

Ensifentrine (Ensifentrinum) is a PDE3 4 inhibitor, although its affinity for PDE3(IC50: 0.4 nM) is 3,440 times higher than that for PDE4(IC50: 1479 nM), that is under clinical development for the treatment of asthma and COPD and, potentially, cystic fibrosis.

Tauroursodeoxycholic-2,2,3,4,4-d5 Acid is a deuterated compound of Tauroursodeoxycholic Acid. Tauroursodeoxycholic Acid has a CAS number of 14605-22-2. Tauroursodeoxycholate, also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

P552-02, also known as KM-003, PS 552-02, or 552-02, is a sodium channel blocker potentially for the treatment of cystic fibrosis.

Tezacaftor-d4 is a deuterated compound of Tezacaftor. Tezacaftor has a CAS number of 1152311-62-0. VX-661 is a small molecule that can be used as a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) gene function.

Tauroursodeoxycholic Acid-d4 Sodium Salt is a deuterated compound of Tauroursodeoxycholic Acid Sodium Salt. Tauroursodeoxycholic Acid Sodium Salt has a CAS number of 14605-22-2. Tauroursodeoxycholate, also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

Ivacaftor hydrate is an orally bioavailable CFTR potentiator. It also is used for cystic fibrosis treatment.

(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].

Ganglioside GM2 asialo (asialo- GM2) is a glycosphingolipid containing three monosaccharide residues and a fatty acid of variable chain length but lacking the sialic acid residue present on ganglioside M2. Asialo-GM2 levels are low-to-undetectable in normal human brain, but it accumulates in the brain of patients with Tay-Sachs and Sandhoff disease, which are neurodegenerative disorders characterized by deficiency of lysosomal β-hexosaminidase A and B, respectively. It also binds to various bacteria, including Pseudomonas isolates derived from cystic fibrosis patients. Asialo-GM2 mixture contains ganglioside GM2 asialo molecular species with fatty acyl chains of variable lengths.

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].

(Rac)-Tezacaftor ((Rac)-VX-661) is a racemic mixture of the F508del CFTR corrector, Tezacaftor, used in cystic fibrosis research [1].

PAD-IN-2, a potent inhibitor of PAD4 with an IC50 value <1 μM, can be utilized to study autoimmune diseases and cancers, including rheumatoid arthritis, vasculitis, systemic lupus erythematosus, cutaneous lupus erythematosus, cystic fibrosis, ulcerative colitis, asthma, multiple sclerosis, and psoriasis.

Lonodelestat TFA (POL6014 TFA) is a potent and selective peptide inhibitor of human neutrophil elastase (hNE) with demonstrated oral activity, showing promise for cystic fibrosis (CF) research [1] [2] [3].

CTP-656, a cystic fibrosis transmembrane conductance regulator (CFTR) channel activator, is used potentially for the treatment of cystic fibrosis.

Navocaftor, as a cystic fibrosis transmembrane regulator ( CFTR ), is a protein modulator (US 20200377491 Al, example 1).

In epididymal epithelial cells, AF-2785 inhibits cystic fibrosis transmembrane conductance regulator Cl(-) channels.

KM-003, a sodium channel blocker, is used potentially for the treatment of cystic fibrosis.

C4 Ceramide is a bioactive sphingolipid and cell-permeable analog of naturally occurring ceramides. [1] [2] [3] It inhibits IL-4 production by 16% in EL4 T cells stimulated with phorbol 12-myristate 13-acetate when used at a concentration of 10 μM. [1] C4 Ceramide is cytotoxic to SK-BR-3 and MCF-7/Adr breast cancer cells (IC50s = 15.9 and 19.9 μM, respectively). [2] C4 Ceramide also increases maturation and stability of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation, enhances cAMP-activated chloride secretion, and suppresses secretion of IL-8 in primary epithelial cells isolated from patients with cystic fibrosis.[3]

SLC26A3-IN-3 (compound 4az), a potent SLC26A3 inhibitor with an IC50 of 40 nM, is utilized for constipation and cystic fibrosis research [1].

Zatonacaftor, a cystic fibrosis transmembrane regulator (CFTR) modulator, is utilized in the research of cystic fibrosis [1] [2].

Duramycin is a cyclic peptide lantibiotic derived from Streptomyces cinnamoneuma. Duramycin interacts with phosphatidylethanolamine (PE) and has antibacterial, antiviral effects. Duramycin stimulates chloride secretion in airway epithelium and has the potential for cystic fibrosis treatment.

Aztreonam lysine is an inhaled anti-pseudomonal treatment for people who have pulmonary Pseudomonas aeruginosa infection with cystic fibrosis (CF).