Targeted Protein Degraders

Targeted protein degradation based on proteolysis-targeting chimeras (PROTACs) is a rapidly developing technology for the inhibition of protein function, via direct degradation mediated by ubiquitin-proteasome system. PROTACs are hetero-bifunctional small molecules capable of bringing the target and the degradation system into contact, thus removing specific proteins from within cells.

This innovative event-driven therapeutic modality has indeed the enormous advantage of focusing on targets considered undruggable from a traditional medicinal chemistry perspective, thus expanding the potential therapeutic strategies to counteract several diseases.

Starting from the discovery of a translation initiation factor involved in the pathological evolution from nonalcoholic fatty liver disease (NAFLD) to nonalcoholic steatohepatitis (NASH) and then to hepatocellular carcinoma (HCC), PROTACs-mediated inhibition should be an effective strategy to counteract the development and the progression of the most widespread form of liver cancer nowadays.

scheme of the project