Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

Nat Commun. 2017 Mar 2:8:14630. doi: 10.1038/ncomms14630.

Abstract

Monoclonal antibodies are one of the fastest growing classes of pharmaceutical products, however, their potential is limited by the high cost of development and manufacturing. Here we present a safe and cost-effective platform for in vivo expression of therapeutic antibodies using nucleoside-modified mRNA. To demonstrate feasibility and protective efficacy, nucleoside-modified mRNAs encoding the light and heavy chains of the broadly neutralizing anti-HIV-1 antibody VRC01 are generated and encapsulated into lipid nanoparticles. Systemic administration of 1.4 mg kg-1 of mRNA into mice results in ∼170 μg ml-1 VRC01 antibody concentrations in the plasma 24 h post injection. Weekly injections of 1 mg kg-1 of mRNA into immunodeficient mice maintain trough VRC01 levels above 40 μg ml-1. Most importantly, the translated antibody from a single injection of VRC01 mRNA protects humanized mice from intravenous HIV-1 challenge, demonstrating that nucleoside-modified mRNA represents a viable delivery platform for passive immunotherapy against HIV-1 with expansion to a variety of diseases.

Publication types

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

MeSH terms

  • Animals
  • Antibodies, Monoclonal / genetics
  • Antibodies, Neutralizing / genetics*
  • Broadly Neutralizing Antibodies
  • Drug Administration Schedule
  • Female
  • HIV Antibodies / biosynthesis
  • HIV Infections / immunology
  • HIV Infections / therapy
  • HIV-1 / drug effects*
  • HIV-1 / immunology
  • Humans
  • Immunization, Passive
  • Lipids / chemistry
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred C57BL
  • Mice, Inbred NOD
  • Mice, SCID
  • Mice, Transgenic
  • Nanoparticles / chemistry
  • Nucleosides / chemistry*
  • RNA, Messenger / administration & dosage*
  • RNA, Messenger / chemistry
  • RNA, Messenger / pharmacology
  • RNA, Messenger / therapeutic use

Substances

  • Antibodies, Monoclonal
  • Antibodies, Neutralizing
  • Broadly Neutralizing Antibodies
  • HIV Antibodies
  • Lipids
  • Nucleosides
  • RNA, Messenger
  • VRC01 monoclonal antibody