Targeted Protein Degradation: Eliminating Disease-Causing Proteins

While DNA captures most of the fanfare, proteins are the catalytic and structural superstars of the cell. However, they can also become problematic. Cells have intricate mechanisms to remove damaged or mis-expressed proteins that could be deleterious to cellular function. This process is mediated by a process called ubiquitination, mediated by a special class of proteins called E3 ligases. Ubiquitin is the tag that's added that signals that a protein should be moved to the biochemical garbage can.  Dr. Juliet WIlliams of Kymera describes how their company has used modeling and A.I. to design molecular linkers that connect a protein that needs to be degraded with the machinery to tag it for destruction. The goal of this line of therapeutics is to target a suite of proteins that need to be degraded for normal health and development. Their pipeline contains multiple clinical and pre-clinical trials, and the approach is an exciting complement to other drug discovery methods.