2. Disease Areas
  3. Respiratory Disease
  4. Pulmonary Disease
  5. Cystic Fibrosis

Cystic Fibrosis

Cystic fibrosis is a progressive, inherited genetic disorder caused by mutations in the CFTR gene, leading to the production of thick, sticky mucus that obstructs the lungs, pancreas, and other organs. This mucus buildup results in chronic respiratory infections, impaired lung function, pancreatic insufficiency, digestive problems, and malnutrition. The disease affects multiple systems, with common symptoms including coughing, wheezing, recurrent sinus infections, clubbing of the fingers, and male infertility. Although cystic fibrosis is life-limiting, advancements in treatment—including airway clearance techniques, enzyme replacement therapy, medications targeting the underlying defect, and nutritional support—have significantly improved survival and quality of life. It is inherited in an autosomal recessive pattern and is the most common fatal genetic disease among people of European descent, affecting approximately 40,000 individuals worldwide.

Cystic Fibrosis (11):

Cat. No. Product Name CAS No. Purity Chemical Structure
  • HY-114231B
    Exaluren disulfate 2244622-33-9 ≥98.0%
    Exaluren (ELX-02; NB-124) disulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren disulfate is used for the research of cystic fibrosis caused by nonsense mutations.
    Exaluren disulfate
  • HY-114231
    Exaluren 1375073-93-0 98.0%
    Exaluren (ELX-02; NB-124) is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren is used for the research of cystic fibrosis caused by nonsense mutations.
    Exaluren
  • HY-145603A
    (R)-Vanzacaftor 2374124-48-6 98.02%
    (R)-Vanzacaftor ((R)-VX-121) is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator with max activity of 30-60% and EC50 <1 μM in enteroid cells. (R)-Vanzacaftor can be used for the research of cystic fibrosis.
    (R)-Vanzacaftor
  • HY-119229
    VRT-325 815592-21-3 99.58%
    VRT-325 is a CFTR modulator. VRT-325 inhibits disulfide cross-linking between cysteines in transmembrane segments 6 and 7 of CFTR and P-gp. VRT-325 promotes maturation of CFTR and P-gp processing mutants, rescues ΔF508-CFTR folding at the endoplasmic reticulum. VRT-325 binds ΔF508-CFTR nucleotide-binding domain 1, and increases mature ΔF508-CFTR cell surface expression and chloride conductance. VRT-325 can be used for the research of cystic fibrosis[1][3].
    VRT-325
  • HY-W984122
    CFTR corrector 17 912790-04-6 98.05%
    CFTR corrector 17 is an isoxazole compound and CFTR modulator. CFTR corrector 17 can be used for the research of cystic fibrosis.
    CFTR corrector 17
  • HY-W403607
    CBIQ 32081-28-0 99.94%
    CBIQ (4-Chlorobenzo[f]isoquinoline) is a benzoisoquinoline compound. CBIQ can activate the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- ion channels and the intermediate-conductance calcium-sensitive K+ channel (KCNN4) with Kd values of 0.1 and 3.9 μM. CBIQ can be used for the research related to cystic fibrosis.
    CBIQ
  • HY-183725
    MS5128 3060520-82-0
    MS5128 is an OTUB1-based deubiquitinase-targeting chimera (DUBTAC) targeting CFTR. MS5128 recruits OTUB1 to CFTR to induce deubiquitination and stabilization of CFTR. MS5128 can be used for the research of cystic fibrosis. (Blue: CFTR Ligand (HY-183724); Pink: OTUB1 Ligand (HY-13262); Black: Linker)
    MS5128
  • HY-W004782
    3-Methylthiophene-2-carboxylic acid 23806-24-8 99.93%
    3-Methylthiophene-2-carboxylic acid (compound 3) is a potent Pseudomonas quinolone signal system protein E (PqsE) inhibitor with a Kd of 19.6 µM and an IC50 of 40 µM. 3-Methylthiophene-2-carboxylic acid binds to the active center of PqsE and shows inhibition of the thioesterase activity. Methylthiophene-2-carboxylic acid can permeate into P. aeruginosa and affect 2,4-dihydroxyquinoline (DHQ) levels to a similar extent as deletion of the PqsE gene. 3-Methylthiophene-2-carboxylic acid can be used for cystic fibrosis research.
    3-Methylthiophene-2-carboxylic acid
  • HY-183724
    MS8572 3060517-63-4
    MS8572 is a selective OTUB1 covalent ligand. MS8572 covalently modifies the noncatalytic cysteine 23 residue of OTUB1 and does not inhibit OTUB1 deubiquitinase activity. MS8572 can be used for development of deubiquitinase-targeting chimeras for targeted protein stabilization.
    MS8572
  • HY-185425
    SM6.1-azide 2320477-40-3
    SM6.1-azide (Compound 2a) is a ligand for αvβ6 integrin with a azide group. The azide group of SM6.1-azide can undergo a click chemical reaction with small molecules, peptides or siRNA containing DBCO or Alkyne groups, enabling pulmonary-targeted delivery .
    SM6.1-azide
  • HY-183001
    ATX-012 1777792-44-5
    ATX-012 is an ionizable cationic lipid. ATX-012 can be used to prepare lipid formulations that encapsulate CFTR mRNA. ATX-012 is applicable to research related to cystic fibrosis.
    ATX-012