Kappa-on-Heavy (KoH) bodies are a distinct class of fully-human antibody-like therapeutic agents with antigen-binding properties

Proc Natl Acad Sci U S A. 2020 Jan 7;117(1):292-299. doi: 10.1073/pnas.1901734117. Epub 2019 Dec 26.

Abstract

We describe a Kappa-on-Heavy (KoH) mouse that produces a class of highly diverse, fully human, antibody-like agents. This mouse was made by replacing the germline variable sequences of both the Ig heavy-chain (IgH) and Ig kappa (IgK) loci with the human IgK germline variable sequences, producing antibody-like molecules with an antigen binding site made up of 2 kappa variable domains. These molecules, named KoH bodies, structurally mimic naturally existing Bence-Jones light-chain dimers in their variable domains and remain wild-type in their antibody constant domains. Unlike artificially diversified, nonimmunoglobulin alternative scaffolds (e.g., DARPins), KoH bodies consist of a configuration of normal Ig scaffolds that undergo natural diversification in B cells. Monoclonal KoH bodies have properties similar to those of conventional antibodies but exhibit an enhanced ability to bind small molecules such as the endogenous cardiotonic steroid marinobufagenin (MBG) and nicotine. A comparison of crystal structures of MBG bound to a KoH Fab versus a conventional Fab showed that the KoH body has a much deeper binding pocket, allowing MBG to be held 4 Å further down into the combining site between the 2 variable domains.

Keywords: alternative binding scaffolds; immunoglobulin loci; therapeutic antibodies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Antibodies / chemistry*
  • Antibodies / genetics
  • Antibodies / immunology*
  • Antibodies / therapeutic use
  • Antigens / immunology*
  • Base Sequence
  • Binding Sites, Antibody / genetics
  • Bufanolides
  • Genetic Engineering
  • Humans
  • Immunoglobulin Heavy Chains / chemistry*
  • Immunoglobulin Heavy Chains / genetics
  • Immunoglobulin Variable Region / chemistry*
  • Immunoglobulin Variable Region / genetics
  • Immunoglobulin Variable Region / immunology*
  • Immunoglobulin kappa-Chains / chemistry*
  • Immunoglobulin kappa-Chains / genetics
  • Mice
  • Models, Molecular
  • Nicotine
  • Protein Conformation

Substances

  • Antibodies
  • Antigens
  • Bufanolides
  • Immunoglobulin Heavy Chains
  • Immunoglobulin Variable Region
  • Immunoglobulin kappa-Chains
  • marinobufagenin
  • Nicotine

Associated data

  • PDB/6PYC
  • PDB/6PYD