[PDF][PDF] Targeted protein degradation: from chemical biology to drug discovery

PM Cromm, CM Crews - Cell chemical biology, 2017 - cell.com
PM Cromm, CM Crews
Cell chemical biology, 2017cell.com
Traditional pharmaceutical drug discovery is almost exclusively focused on directly
controlling protein activity to cure diseases. Modulators of protein activity, especially
inhibitors, are developed and applied at high concentration to achieve maximal effects.
Thereby, reduced bioavailability and off-target effects can hamper compound efficacy.
Nucleic acid-based strategies that control protein function by affecting expression have
emerged as an alternative. However, metabolic stability and broad bioavailability represent …
Traditional pharmaceutical drug discovery is almost exclusively focused on directly controlling protein activity to cure diseases. Modulators of protein activity, especially inhibitors, are developed and applied at high concentration to achieve maximal effects. Thereby, reduced bioavailability and off-target effects can hamper compound efficacy. Nucleic acid-based strategies that control protein function by affecting expression have emerged as an alternative. However, metabolic stability and broad bioavailability represent development hurdles that remain to be overcome for these approaches. More recently, utilizing the cell's own protein destruction machinery for selective degradation of essential drivers of human disorders has opened up a new and exciting area of drug discovery. Small-molecule-induced proteolysis of selected substrates offers the potential of reaching beyond the limitations of the current pharmaceutical paradigm to expand the druggable target space.
cell.com