Compound-13-(GSK716)

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…


 become a member to continue reading

Ask your company or librarian about a Drug Hunter membership to to see all summaries and get access to even more helpful information.

  • Stay up-to-date and save more time with exclusive members-only content
  • On-board new team members with educational content and case studies
  • Generate ideas and discussion with easy-to-read slide decks
  • Generate ideas and discussion with easy-to-read slide decks