mutant alpha-1-antitrypsin folding corrector

sub-µM in cell, high predicted human %F

from 2T component DEL + SBDD

Bioorg. Med. Chem. Lett., Mar. 19, 2021

GlaxoSmithKline, Stevenage, UK / UCL

The GlaxoSmithKline (GSK) Z α1-antitrypsin stabilizer (“compound 13,” GSK716) acts by blocking the formation of polymers within the endoplasmic reticulum of hepatocytes for treatment of α1-antitrypsin deficiency. It was…

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