A Kinetic Scout Approach Accelerates Targeted Protein Degrader Development

被引:0
作者
Fan, Angela T. [1 ]
Gadbois, Gillian E. [1 ]
Huang, Hai-Tsang [2 ,4 ]
Chaudhry, Charu [8 ]
Jiang, Jiewei [1 ]
Sigua, Logan H. [3 ]
Smith, Emily R. [1 ]
Wu, Sitong [1 ]
Poirier, Grace J. [1 ]
Dunne-Dombrink, Kara [1 ]
Goyal, Pavitra [1 ]
Tao, Andrew J. [1 ]
Sellers, William R. [2 ,4 ]
Fischer, Eric S. [5 ,6 ]
Donovan, Katherine A. [5 ,6 ]
Ferguson, Fleur M. [1 ,7 ]
机构
[1] Univ Calif San Diego, Dept Chem & Biochem, San Diego, CA 92110 USA
[2] Broad Inst Harvard & MIT, Cambridge, MA USA
[3] Univ Calif San Diego, Med Scientist Training Program, San Diego, CA USA
[4] Harvard Med Sch, Dana Farber Canc Inst, Dept Med Oncol, Boston, MA USA
[5] Dana Farber Canc Inst, Dept Canc Biol, Boston, MA USA
[6] Harvard Med Sch, Dept Biol Chem & Mol Pharmacol, Boston, MA USA
[7] Univ Calif San Diego, Skaggs Sch Pharm & Pharmaceut Sci, San Diego, CA 92110 USA
[8] J&J Innovat Med, Discovery Technol Mol Pharmacol, Spring House, PA 19477 USA
关键词
Targeted protein degradation; PROTACs; compound residence time; proteomics; proteasomal degradation;
D O I
10.1002/anie.202417272
中图分类号
O6 [化学];
学科分类号
0703 ;
摘要
Bifunctional molecules such as targeted protein degraders induce proximity to promote gain-of-function pharmacology. These powerful approaches have gained broad traction across academia and the pharmaceutical industry, leading to an intensive focus on strategies that can accelerate their identification and optimization. We and others have previously used chemical proteomics to map degradable target space, and these datasets have been used to develop and train multiparameter models to extend degradability predictions across the proteome. In this study, we now turn our attention to develop generalizable chemistry strategies to accelerate the development of new bifunctional degraders. We implement lysine-targeted reversible-covalent chemistry to rationally tune the binding kinetics at the protein-of-interest across a set of 25 targets. We define an unbiased workflow consisting of global proteomics analysis, IP/MS of ternary complexes and the E-STUB assay, to mechanistically characterize the effects of ligand residence time on targeted protein degradation and formulate hypotheses about the rate-limiting step of degradation for each target. Our key finding is that target residence time is a major determinant of degrader activity, and this can be rapidly and rationally tuned through the synthesis of a minimal number of analogues to accelerate early degrader discovery and optimization.
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页数:10
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