Through targeted protein degradation, we can address disease by controlling the amount of a harmful protein rather than trying to change or inhibit its function.
The control of protein levels is accomplished with a small molecule drug called a degrader.1, 2 This degrader takes advantage of a natural cellular process called ubiquitination. Ubiquitination is the first step of a versatile, widely-conserved, and modular system used by cells to recycle proteins. The ultimate step in the overall process is the destruction of the protein by the proteasome.