Targeted protein degradation in vivo with Proteolysis Targeting Chimeras: Current status and future considerations
Tài liệu tham khảo
Bartlett, 2004, The evolution of thalidomide and its IMiD derivatives as anticancer agents, Nat Rev Cancer, 4, 314, 10.1038/nrc1323
Ito, 2016, Cereblon and its downstream substrates as molecular targets of immunomodulatory drugs, Int J Hematol, 104, 293, 10.1007/s12185-016-2073-4
Patel, 2018, Selective estrogen receptor modulators (SERMs) and selective estrogen receptor degraders (SERDs) in cancer treatment, Pharmacol Ther, 186, 1, 10.1016/j.pharmthera.2017.12.012
Lai, 2017, Induced protein degradation: an emerging drug discovery paradigm, Nat Rev Drug Discov, 16, 101, 10.1038/nrd.2016.211
Raina, 2017, Targeted protein knockdown using small molecule degraders, Curr Opin Chem Biol, 39, 46, 10.1016/j.cbpa.2017.05.016
Neklesa, 2017, Targeted protein degradation by PROTACs, Pharmacol Ther, 174, 138, 10.1016/j.pharmthera.2017.02.027
An, 2018, Small-molecule PROTACs: an emerging and promising approach for the development of targeted therapy drugs, EBioMed, 36, 553, 10.1016/j.ebiom.2018.09.005
Tinworth, 2016, Small molecule-mediated protein knockdown as a new approach to drug discovery Med, Chem Commun, 7, 2206, 10.1039/C6MD00347H
Hines, 2018, MDM2-recruiting PROTAC offers superior, synergistic anti-proliferative activity via simultaneous degradation of BRD4 and stabilization of p53, Cancer Res, 79, 251, 10.1158/0008-5472.CAN-18-2918
Floris, 2018, Genetic-driven druggable target identification and validation, Trends Genet, 34, 558, 10.1016/j.tig.2018.04.004
Mullard, 2017, An audience with Sean Harper, Nat Rev Drug Discov, 17, 10
Nelson, 2015, The support of human genetic evidence for approved drug indications, Nat Genet, 47, 856, 10.1038/ng.3314
Kung, 2016, Structural basis for the non-catalytic functions of protein kinases, Structure, 24, 7, 10.1016/j.str.2015.10.020
Knight, 2007, Chemical genetics: where genetics and pharmacology meet, Cell, 128, 425, 10.1016/j.cell.2007.01.021
Eisener-Dorman, 2009, Cautionary insights on knockout mouse studies: the gene or not the gene?, Brain Behav Immun, 23, 318, 10.1016/j.bbi.2008.09.001
Bassi, 2018, Modulating PCAF/GCN5 Immune Cell Function through a PROTAC approach, ACS Chem Biol, 13, 2862, 10.1021/acschembio.8b00705
Cromm, 2018, Addressing kinase-independent functions of fak via PROTAC-mediated degradation, J Am Chem Soc, 140, 17019, 10.1021/jacs.8b08008
Tai, 2015, “Emerging Roles of Focal Adhesion Kinase in Cancer,”, BioMed Research International, 2015, 13, 10.1155/2015/690690
Saenz, 2017, Novel BET protein proteolysis-targeting chimera exerts superior lethal activity than bromodomain inhibitor (BETi) against post-myeloproliferative neoplasm secondary (s) AML cells, Leukemia, 31, 1951, 10.1038/leu.2016.393
Sun, 2018, BET protein proteolysis targeting chimera (PROTAC) exerts potent lethal activity against mantle cell lymphoma cells, Leukemia, 32, 343, 10.1038/leu.2017.207
Zorba, 2018, Delineating the role of cooperativity in the design of potent PROTACs for BTK, Proc Natl Acad Sci U S A, 115, E7285, 10.1073/pnas.1803662115
Tuntland, 2014, Implementation of pharmacokinetic and pharmacodynamic strategies in early research phases of drug discovery and development at Novartis Institute of Biomedical Research, Front Pharmacol, 5, 174, 10.3389/fphar.2014.00174
Cook, 2014, Lessons learned from the fate of AstraZeneca’s drug pipeline: a five-dimensional framework, Nat Rev Drug Discov, 13, 419, 10.1038/nrd4309
Morgan, 2012, Can the flow of medicines be improved? Fundamental pharmacokinetic and pharmacological principles toward improving phase II survival, Drug Discov Today, 17, 419, 10.1016/j.drudis.2011.12.020
Bondeson, 2015, Catalytic in vivo protein knockdown by small-molecule PROTACs, Nat Chem Biol, 11, 611, 10.1038/nchembio.1858
Winter, 2015, DRUG DEVELOPMENT. Phthalimide conjugation as a strategy for in vivo target protein degradation, Science, 348, 1376, 10.1126/science.aab1433
Ohoka, 2017, In vivo knockdown of pathogenic proteins via specific and nongenetic inhibitor of apoptosis protein (IAP)-dependent protein erasers (SNIPERs), J Biol Chem, 292, 4556, 10.1074/jbc.M116.768853
Casillas, 2017, Conjugates comprising RIPK2 inhibitors, WO2017/182418 A1
Flanagan, 2017
Neklesa, 2018
Qin, 2018, Discovery of QCA570 as an exceptionally potent and efficacious proteolysis targeting chimera (PROTAC) degrader of the bromodomain and extra-terminal (BET) proteins capable of inducing complete and durable tumor regression, J Med Chem, 61, 6685, 10.1021/acs.jmedchem.8b00506
Zhang, 2018, Protein targeting chimeric molecules specific for bromodomain and extra-terminal motif family proteins are active against pre-clinical models of multiple myeloma, Leukemia, 32, 2224, 10.1038/s41375-018-0044-x
Zhou, 2018, Discovery of a small-molecule degrader of bromodomain and extra-terminal (BET) proteins with picomolar cellular potencies and capable of achieving tumor regression, J Med Chem, 61, 462, 10.1021/acs.jmedchem.6b01816
Dobrovolsky, 2018, Bruton’s tyrosine kinase degradation as a therapeutic strategy for cancer, Blood, 133, 952, 10.1182/blood-2018-07-862953
Li, 2018, Discovery of MD-224 as a first-in-class, highly potent, and efficacious proteolysis targeting chimera murine double minute 2 degrader capable of achieving complete and durable tumor regression, J Med Chem, 62, 448, 10.1021/acs.jmedchem.8b00909
Raina, 2016, PROTAC-induced BET protein degradation as a therapy for castration-resistant prostate cancer, Proc Natl Acad Sci U S A, 13, 7124, 10.1073/pnas.1521738113
Jiang, 2018, Development of stabilized peptide-based PROTACs against estrogen receptor alpha, ACS Chem Biol, 13, 628, 10.1021/acschembio.7b00985
Kang, 2018, Induced protein degradation of anaplastic lymphoma kinase (ALK) by proteolysis targeting chimera (PROTAC), Biochem Biophys Res Commun, 505, 542, 10.1016/j.bbrc.2018.09.169
Burslem, 2018, Enhancing antiproliferative activity and selectivity of a FLT-3 inhibitor by proteolysis targeting chimera conversion, J Am Chem Soc, 140, 16428, 10.1021/jacs.8b10320
Ohoka, 2017, In vivo knockdown of pathogenic proteins via specific and nongenetic inhibitor of apoptosis protein (IAP)-dependent protein erasers (SNIPERs), J Biol Chem, 292, 4556, 10.1074/jbc.M116.768853
Danhof, 2005, Mechanism-based pharmacokinetic-pharmacodynamic modeling-a new classification of biomarkers, Pharm Res, 22, 1432, 10.1007/s11095-005-5882-3
Chen, 2018, Impact of dosing schedule in animal experiments on compound progression decisions, Drug Discovery Today, 24, 371, 10.1016/j.drudis.2018.11.006
Gabrielsson, 2018, Lost in translation: What’s in an EC50? Innovative PK/PD reasoning in the drug development context, Eur J Pharmacol, 835, 154, 10.1016/j.ejphar.2018.07.037
Dowty, 2014, Preclinical to clinical translation of tofacitinib, a janus kinase inhibitor, in rheumatoid arthritis, J Pharmacol Exp Ther, 348, 165, 10.1124/jpet.113.209304
Churcher, 2018, Protac-induced protein degradation in drug discovery: breaking the rules or just making new ones?, J Med Chem, 61, 444, 10.1021/acs.jmedchem.7b01272
Mager, 2006, Target-mediated drug disposition and dynamics, Biochem Pharmacol, 72, 1, 10.1016/j.bcp.2005.12.041
Mager, 2003, Diversity of mechanism-based pharmacodynamic models, Drug Metab Dispos, 31, 510, 10.1124/dmd.31.5.510
Kola, 2004, Can the pharmaceutical industry reduce attrition rates?, Nat Rev Drug Discov, 3, 711, 10.1038/nrd1470