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Tezacaftor CAS# 1152311-62-0 替扎卡托

Tezacaftor, also known asVX-661, is CFTR modulator. 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.
📌 参考资料/链接:
Reference: Dhamankar, Varsha; Dinehart, Kirk Raymond; Dokou, Eleni; Ferris, Lori Ann; Gopinathan, Nishanth; McCarty, Katie; Metzler, Catherine; Zhang, Beili. Preparation of cystalline forms and pharmaceutical compositions comprising them as CFTR modulators for the treatment of cystic fibrosis. Assignee Vertex Pharmaceuticals Incorporated, USA. WO 2019079760. (2019).

📄 详细内容

CAS No. 1152311-62-0 替扎卡托Tezacaftor synthetic routes


Dhamankar, Varsha; Dinehart, Kirk Raymond; Dokou, Eleni; Ferris, Lori Ann; Gopinathan, Nishanth; McCarty, Katie; Metzler, Catherine; Zhang, Beili. Preparation of cystalline forms and pharmaceutical compositions comprising them as CFTR modulators for the treatment of cystic fibrosis. Assignee Vertex Pharmaceuticals Incorporated, USA. WO 2019079760. (2019).
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