Discovery of three small-molecule inhibitors targeting Ebolavirus genome replication and transcription

J Gen Virol. 2025 Dec;106(12):002183. doi: 10.1099/jgv.0.002183.

Abstract

The 2013 Ebola virus (EBOV) outbreak was the largest in history. Despite recent advances in both vaccines and monoclonal antibody therapies, 12 years later, EBOV still poses a substantial threat. Previously, we published a ligand discovery pipeline combining in silico screening of compounds with a robust and rapid EBOV minigenome assay for early-stage inhibitor validation at Biological Safety Level 2. Here, we present the further use of this pipeline to identify three compounds that also inhibit EBOV minigenome transcription and replication. They are efficacious in the nM range, exhibited low cytotoxicity and were specific, with no effect on either a T7 RNA polymerase-driven firefly luciferase or a Bunyamwera virus minigenome. Furthermore, these small-molecule inhibitors exhibited the ability to block EBOV minigenome activity when applied after establishment of replication complexes, with implications for potential post-exposure EBOV treatment.

Keywords: Ebolavirus (EBOV); drug discovery; minigenome; nucleoprotein (NP); small-molecule inhibitors (SMIs).

MeSH terms

  • Animals
  • Antiviral Agents* / chemistry
  • Antiviral Agents* / isolation & purification
  • Antiviral Agents* / pharmacology
  • Drug Discovery
  • Ebolavirus* / drug effects
  • Ebolavirus* / genetics
  • Ebolavirus* / physiology
  • Genome, Viral* / drug effects
  • Hemorrhagic Fever, Ebola / drug therapy
  • Hemorrhagic Fever, Ebola / virology
  • Humans
  • Small Molecule Libraries* / pharmacology
  • Transcription, Genetic* / drug effects
  • Virus Replication* / drug effects

Substances

  • Antiviral Agents
  • Small Molecule Libraries