Conditionally Activatable Chimeras for Tumor-Specific Membrane Protein Degradation

J Am Chem Soc. 2024 Dec 4;146(48):32933-32941. doi: 10.1021/jacs.4c06160. Epub 2024 Nov 19.

Abstract

The recent advancements on membrane protein degraders (MPDs) have broadened the applicability of proteolysis-targeting chimeras (PROTACs) beyond intracellular proteins to include the previously "undruggable" cell-surface targets. However, the potential toxicity of MPDs caused by undesired off-target degradation poses a significant challenge to clinical deployment, mirroring concerns associated with PROTACs. Here, we introduce a conditionally activatable membrane protein degrader (Pro-MPD), which leverages the specificity and high affinity of biparatopic nanobodies combined with a tumor microenvironment-activated cell-penetrating peptide (Pro-CPP) to achieve on-target activated internalization and degradation of PD-L1 within tumor sites. This modularly designed Pro-MPD demonstrated a high target degradation efficiency and T cell reactivation, as well as sustained inhibition of tumor growth in xenograft models, highlighting its potential as a safer and highly efficient MPD for in vivo applications. Our work provides a general strategy for the development of conditionally activatable MPDs, which offers a new avenue for reducing the undesired systemic toxicity of MPDs due to the off-tumor degradation.

MeSH terms

  • Animals
  • B7-H1 Antigen / chemistry
  • B7-H1 Antigen / metabolism
  • Cell Line, Tumor
  • Cell-Penetrating Peptides / chemistry
  • Cell-Penetrating Peptides / metabolism
  • Cell-Penetrating Peptides / pharmacology
  • Humans
  • Membrane Proteins / chemistry
  • Membrane Proteins / metabolism
  • Mice
  • Neoplasms / drug therapy
  • Neoplasms / metabolism
  • Neoplasms / pathology
  • Proteolysis* / drug effects
  • Single-Domain Antibodies / chemistry
  • Single-Domain Antibodies / pharmacology
  • Tumor Microenvironment / drug effects

Substances

  • Membrane Proteins
  • Cell-Penetrating Peptides
  • B7-H1 Antigen
  • Single-Domain Antibodies