Perspectives on the development of first-in-class protein degraders

Future Med Chem. 2021 Jul;13(14):1203-1226. doi: 10.4155/fmc-2021-0033. Epub 2021 May 21.

Abstract

Targeted protein degradation is a broad and expanding field aimed at the modulation of protein homeostasis. A focus of this field has been directed toward molecules that hijack the ubiquitin proteasome system with heterobifunctional ligands that recruit a target protein to an E3 ligase to facilitate polyubiquitination and subsequent degradation by the 26S proteasome. Despite the success of these chimeras toward a number of clinically relevant targets, the ultimate breadth and scope of this approach remains uncertain. Here we highlight recent advances in assays and tools available to evaluate targeted protein degradation, including and beyond the study of E3-targeted chimeric ligands. We note several challenges associated with degrader development and discuss various approaches to expanding the protein homeostasis toolbox.

Keywords: autophagy/lysosome targeting; degrader toolbox; drug discovery; targeted protein degradation; ubiquitin proteasome system.

Publication types

  • Review

MeSH terms

  • Autophagy / drug effects
  • Drug Discovery
  • Humans
  • Lysosomes / metabolism
  • Proteasome Endopeptidase Complex / chemistry
  • Proteasome Endopeptidase Complex / metabolism*
  • Proteolysis* / drug effects
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology
  • Ubiquitin / antagonists & inhibitors
  • Ubiquitin / metabolism*
  • Ubiquitin-Protein Ligases / chemistry
  • Ubiquitin-Protein Ligases / metabolism
  • Ubiquitination

Substances

  • Small Molecule Libraries
  • Ubiquitin
  • Ubiquitin-Protein Ligases
  • Proteasome Endopeptidase Complex