Site-Specific Bioconjugation Using SMARTag® Technology: A Practical and Effective Chemoenzymatic Approach to Generate Antibody-Drug Conjugates

Methods Mol Biol. 2019:2033:131-147. doi: 10.1007/978-1-4939-9654-4_10.

Abstract

As a critical feature of the next generation of antibody-drug conjugates (ADCs), site-specific bioconjugation approaches can help to optimize stability, pharmacokinetics, efficacy, and safety as well as improve manufacturing consistency. The SMARTag® technology platform offers a practical and efficient chemoenzymatic solution for site-specific protein modifications. A bioorthogonal aldehyde handle is introduced through the oxidation of a cysteine residue, embedded in a specific peptide sequence (CxPxR), to the aldehyde-bearing formylglycine (fGly). This enzymatic modification is carried out by the formylglycine-generating enzyme (FGE). The broad recognition of this short sequence by FGE within the context of heterologous proteins allows for the introduction of fGly residues at chosen sites in proteins expressed in prokaryotic and eukaryotic systems. The protocol presented here describes the methods for expressing fGly-containing antibodies in eukaryotic cells and subsequent site-specific conjugation with a payload-linker using aldehyde-specific Hydrazino-Iso-Pictet-Spengler (HIPS) chemistry.

Keywords: Antibody–drug conjugate (ADC); Formylglycine (fGly); Formylglycine-generating enzyme (FGE); HIPS; SMARTag®; Site-specific bioconjugation.

MeSH terms

  • Aldehydes / chemistry
  • Glycine / analogs & derivatives
  • Humans
  • Immunoconjugates / chemistry
  • Immunoconjugates / genetics*
  • Oxidoreductases Acting on Sulfur Group Donors / chemistry*
  • Oxidoreductases Acting on Sulfur Group Donors / genetics
  • Peptides / chemistry
  • Peptides / genetics
  • Protein Engineering / methods*
  • Protein Processing, Post-Translational / genetics
  • Proteins / chemistry*
  • Proteins / genetics

Substances

  • Aldehydes
  • Immunoconjugates
  • Peptides
  • Proteins
  • N-formylglycine
  • Oxidoreductases Acting on Sulfur Group Donors
  • SUMF1 protein, human
  • Glycine