| Identification | Back Directory | [Name]
4-Quinolinecarboxylic acid, 8-methyl-2-(3-methyl-2-benzofuranyl)-5-[(1S)-1-(tetrahydro-2H-pyran-4-yl)ethoxy]-, sodium salt (1:1) | [CAS]
2095064-09-6 | [Synonyms]
(R)-Posenacaftor sodium
4-Quinolinecarboxylic acid, 8-methyl-2-(3-methyl-2-benzofuranyl)-5-[(1S)-1-(tetrahydro-2H-pyran-4-yl)ethoxy]-, sodium salt (1:1) | [Molecular Formula]
C27H28NNaO5 | [MOL File]
2095064-09-6.mol | [Molecular Weight]
469.51 |
| Hazard Information | Back Directory | [Uses]
(R)-Posenacaftor (R)-PTI-801) sodium is the R enantiomer of Posenacaftor. Posenacaftor is a cystic fibrosis transmembrane regulator (CFTR) protein modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor is used for the research of cystic fibrosis (CF)[1]. | [Biological Activity]
(R)-Posenacaftor (R)-PTI-801) sodium is the R enantiomer of Posenacaftor. Posenacaftor is a cystic fibrosis transmembrane regulator (CFTR) protein modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor is used for the research of cystic fibrosis (CF)[1].
Cystic fibrosis (CF) is an autosomal recessive disorder, caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR)[1].CFTR is a cAMP-regulated chloride channel that is primarily located at the apical membrane of epithelial cells. Mutations in the CFTR gene lead to the production of a defective and misfolded CFTR protein, and impairs the flow of ions in and out of cells[1].Posenacaftor is a CFTR corrector, correctors are designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways[2]. | [References]
[1]. Benjamin Kopp, et al. Compositions et procédés pour améliorer la fonction cftr dans des cellules affectées par la fibrose kystique. Patent WO2019156946. |
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