product name VX-661
Description: VX-661, also known as Tezacaftor, is a second F508del CFTR corrector and is believed to help CFTR protein reach the cell surface. VX-661 is potentially useful for treatment of cystic fibrosis disease. Cystic fibrosis (CF) is a genetic disease caused by defects in the CF transmembrane regulator (CFTR) gene, which encodes an epithelial chloride channel. The most common mutation, Δ508CFTR, produces a protein that is misfolded and does not reach the cell membrane. VX-661 can correct trafficking of Δ508CFTR and partially restore chloride channel activity.
References: Sci Transl Med. 2014 Jul 23;6(246):246ra97.
520.5
Formula
C26H27F3N2O6
CAS No.
1152311-62-0
Storage
-20℃ for 3 years in powder form
-80℃ for 2 years in solvent
Solubility (In vitro)
DMSO: 100 mg/mL (192.1 mM)
Water: <1 mg/mL
Ethanol: <1 mg/mL
Solubility (In vivo)
Synonyms
other peoduct :References PubMed ID：:http://www.ncbi.nlm.nih.gov/pubmed/19406680
| In Vitro |
In vitro activity: VX-661, known as a CTFR corrector, allows F508del mutant channels to escape degradation and transit to the cell membrane. Kinase Assay: Cell Assay: VX-661 treated alone or in combination with ivacaftor have shown to enhance F508del-CFTR trafficking to the cell surface. VX-661 has been at phase 2 study |
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| In Vivo (Human) | |
| Animal model | |
| Formulation & Dosage | |
| References | Sci Transl Med. 2014 Jul 23;6(246):246ra97. |
