Structure-Activity Relationship of Antibody-Oligonucleotide Conjugates: Evaluating Bioconjugation Strategies for Antibody-Phosphorodiamidate Morpholino Oligomer Conjugates for Drug Development

J Med Chem. 2024 Sep 12;67(17):14868-14884. doi: 10.1021/acs.jmedchem.4c00803. Epub 2024 Aug 28.

Abstract

Antibody-oligonucleotide conjugates (AOCs) are promising treatments for Duchenne muscular dystrophy (DMD). They work via induction of exon skipping and restoration of dystrophin protein in skeletal and heart muscles. The structure-activity relationships (SARs) of AOCs comprising antibody-phosphorodiamidate morpholino oligomers (PMOs) depend on several aspects of their component parts. We evaluate the SAR of antimouse transferrin receptor 1 antibody (αmTfR1)-PMO conjugates: cleavable and noncleavable linkers, linker location on the PMO, and the impact of drug-to-antibody ratios (DARs) on plasma pharmacokinetics (PK), oligonucleotide delivery to tissues, and exon skipping. AOCs containing a stable linker with a DAR9.7 were the most effective PMO delivery vehicles in preclinical studies. We demonstrate that αmTfR1-PMO conjugates induce dystrophin protein restoration in the skeletal and heart muscles of mdx mice. Our results show that αmTfR1-PMO conjugates are a potentially effective approach for the treatment of DMD.

MeSH terms

  • Animals
  • Drug Development
  • Dystrophin / genetics
  • Dystrophin / metabolism
  • Humans
  • Immunoconjugates / chemistry
  • Immunoconjugates / pharmacokinetics
  • Immunoconjugates / pharmacology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred mdx*
  • Morpholinos* / chemistry
  • Morpholinos* / pharmacokinetics
  • Morpholinos* / pharmacology
  • Muscle, Skeletal / metabolism
  • Muscular Dystrophy, Duchenne* / drug therapy
  • Oligonucleotides / chemistry
  • Oligonucleotides / pharmacokinetics
  • Receptors, Transferrin / immunology
  • Receptors, Transferrin / metabolism
  • Structure-Activity Relationship

Substances

  • Morpholinos
  • Dystrophin
  • Immunoconjugates
  • Oligonucleotides
  • Receptors, Transferrin