(R)-Posenacaftor sodium

• CAT Number: I045642
• CAS Number: 2095064-09-6
• Molecular Formula: C27H26NNaO5
• Molecular Weight: 467.49
• Purity: ≥95%
• Synonyms: sodium;8-methyl-2-(3-methyl-1-benzofuran-2-yl)-5-[(1S)-1-(oxan-4-yl)ethoxy]quinoline-4-carboxylate
• InChI: InChI=1S/C27H27NO5.Na/c1-15-8-9-23(32-17(3)18-10-12-31-13-11-18)24-20(27(29)30)14-21(28-25(15)24)26-16(2)19-6-4-5-7-22(19)33-26;/h4-9,14,17-18H,10-13H2,1-3H3,(H,29,30);/q;+1/p-1/t17-;/m0./s1
• InChIKey: FIMNRWQYZVDFTK-LMOVPXPDSA-M
• SMILES: CC1=C2C(=C(C=C1)OC(C)C3CCOCC3)C(=CC(=N2)C4=C(C5=CC=CC=C5O4)C)C(=O)[O-].[Na+]

(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].

Reference

[1]. Benjamin Kopp, et al. Compositions et procédés pour améliorer la fonction cftr dans des cellules affectées par la fibrose kystique. Patent WO2019156946.