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…

get free samples and a Premium trial

Premium members get access to our library of hundreds of in-depth reviews on key molecules, ten new reviews each month, novel drug approval coverage, drug discovery company updates, and more: