Practical synthesis of a phthalimide-based Cereblon ligand to enable PROTAC development

Bioorg Med Chem Lett. 2016 Nov 1;26(21):5260-5262. doi: 10.1016/j.bmcl.2016.09.048. Epub 2016 Sep 19.

Abstract

The use of small molecules to regulate cellular levels of specific proteins is poised to become a powerful technique in the coming years. Critical to the success of any project utilizing such an approach will be the ability to synthesize libraries of candidate small molecules for testing in cellular systems. Herein, we describe a practical synthesis of a phthalimide-based scaffold, which can be easily diversified to make Cereblon-targeting PROTACs. We demonstrate the effectiveness of this approach by synthesizing a 'PROTAC toolbox' of four amines which can be coupled to inhibitors in a straightforward manner.

Keywords: Cereblon; E3 ligase; PROTACs; Thalidomide.

MeSH terms

  • Adaptor Proteins, Signal Transducing
  • Humans
  • Ligands
  • Peptide Hydrolases / chemistry*
  • Phthalimides / chemical synthesis*
  • Phthalimides / chemistry
  • Proteolysis
  • Ubiquitin-Protein Ligases

Substances

  • Adaptor Proteins, Signal Transducing
  • CRBN protein, human
  • Ligands
  • Phthalimides
  • phthalimide
  • Ubiquitin-Protein Ligases
  • Peptide Hydrolases