De Novo-Designed APC/C Inhibitors Provide a Rationale for Targeting RING-Type E3 Ubiquitin Ligases.

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

Abstract

The ubiquitin system represents an attractive pharmacological target for numerous pathological processes, including cancer and neurodegeneration. RING domain-containing E3 ubiquitin ligases constitute the largest class of ubiquitin enzymes, providing a scaffold for substrate recognition and catalysis. Their shallow groove recognition interfaces involving discontinuous epitopes and a lack of defined binding pockets have largely rendered them undruggable. Inspired by natural RING inhibitors, we have developed a pharmacophore-based strategy for the rational design of peptidomimetics targeting RING domains, and we demonstrate its feasibility by using the macromolecular APC/C complex (anaphase-promoting complex/cyclosome). We designed scaffolds binding to the APC/C RING domain and efficiently inhibiting its activity in vitro. Iterative structure-based design and experimental studies to optimize their chemical stability, permeability, and specificity lead to new hydrocarbon-stapled-based molecules inhibiting APC/C in vitro and in cancer cells. Our results provide a robust rationale for targeting RING-containing enzymes of therapeutic value and promising leads for clinical APC/C inhibition.

Details

Original languageEnglish
Pages (from-to)11468–11483
Number of pages16
JournalJournal of medicinal chemistry
Volume68
Issue number11
Early online date21 May 2025
Publication statusPublished - 12 Jun 2025
Peer-reviewedYes

External IDs

ORCID /0000-0002-5175-9311/work/184884922
unpaywall 10.1021/acs.jmedchem.5c00416
Scopus 105005800473

Keywords

Sustainable Development Goals