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.
This affords many opportunities for differentiated medicines:
A single degrader drug can eliminate multiple disease-causing proteins through proteasome degradation of the target protein. However, an inhibitor drug can only block a single disease-causing protein at a time.
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