Accelerating inhibitor discovery for deubiquitinating enzymes

Nat Commun. 2023 Feb 8;14(1):686. doi: 10.1038/s41467-023-36246-0.

Abstract

Deubiquitinating enzymes (DUBs) are an emerging drug target class of ~100 proteases that cleave ubiquitin from protein substrates to regulate many cellular processes. A lack of selective chemical probes impedes pharmacologic interrogation of this important gene family. DUBs engage their cognate ligands through a myriad of interactions. We embrace this structural complexity to tailor a chemical diversification strategy for a DUB-focused covalent library. Pairing our library with activity-based protein profiling as a high-density primary screen, we identify selective hits against 23 endogenous DUBs spanning four subfamilies. Optimization of an azetidine hit yields a probe for the understudied DUB VCPIP1 with nanomolar potency and in-family selectivity. Our success in identifying good chemical starting points as well as structure-activity relationships across the gene family from a modest but purpose-build library challenges current paradigms that emphasize ultrahigh throughput in vitro or virtual screens against an ever-increasing scope of chemical space.

Publication types

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

MeSH terms

  • Deubiquitinating Enzymes / metabolism
  • Endopeptidases* / metabolism
  • Peptide Hydrolases / metabolism
  • Structure-Activity Relationship
  • Ubiquitin* / metabolism
  • Ubiquitination

Substances

  • Ubiquitin
  • Endopeptidases
  • Peptide Hydrolases
  • Deubiquitinating Enzymes