Antithrombotic effect of antisense factor xi oligonucleotide treatment in primates

Arteriosclerosis, Thrombosis, and Vascular Biology

Volumn 33, Issue 7, 2013, Pages 1670-1678

  Crosby, Jeffrey R ^a   Marzec, Ulla ^b   Revenko, Alexey S ^a   Zhao, Chenguang ^a   Gao, Dacao ^a   Matafonov, Anton ^a,c   Gailani, David ^c   MacLeod, A Robert ^d   Tucker, Erik I ^b,d   Gruber, Andras ^b   Hanson, Stephen R ^b   Monia, Brett P ^a  

^a ISIS PHARMACEUTICALS   (United States)

^b Oregon Health and Science University Department of Biomedical Engineering   (United States)

^c VANDERBILT UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d Aronora Inc   (United States)

Author keywords

Antisense oligonucleotide; Factor XI; Platelets; Thrombus formation; Vascular graft

Indexed keywords

ANTICOAGULANT AGENT; ANTISENSE FACTOR XI OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; BLOOD CLOTTING FACTOR 11; COLLAGEN; MESSENGER RNA; MONOCLONAL ANTIBODY; THROMBIN; UNCLASSIFIED DRUG;

ANIMAL MODEL; ANTICOAGULANT THERAPY; ANTICOAGULATION; ARTICLE; BABOON; BLOOD CLOTTING; BLOOD FLOW; BLOOD LEVEL; CONTROLLED STUDY; DOSE RESPONSE; DRUG SAFETY; HEMOSTASIS; IN VIVO STUDY; MOLECULARLY TARGETED THERAPY; NONHUMAN; PRIORITY JOURNAL; PROTEIN FUNCTION; THROMBOGENICITY;

ANTISENSE OLIGONUCLEOTIDE; FACTOR XI; PLATELETS; THROMBUS FORMATION; VASCULAR GRAFT;

ANIMALS; ANTIBODIES, MONOCLONAL; ARTERIOVENOUS SHUNT, SURGICAL; BLEEDING TIME; BLOOD COAGULATION; COLLAGEN; DISEASE MODELS, ANIMAL; DOSE-RESPONSE RELATIONSHIP, DRUG; DOWN-REGULATION; FACTOR XI; FIBRINOLYTIC AGENTS; HEMORRHAGE; MACACA FASCICULARIS; OLIGONUCLEOTIDES, ANTISENSE; PAPIO; THROMBIN; THROMBOSIS; TIME FACTORS;

EID: 84879076198     PISSN: 10795642     EISSN: 15244636     Source Type: Journal    
DOI: 10.1161/ATVBAHA.113.301282     Document Type: Article

References (31)

1. Davie EW, Fujikawa K, Kisiel W. The coagulation cascade: initiation, maintenance, and regulation. Biochemistry. 1991;30:10363-10370.
2. Macfarlane RG. An enzyme cascade in the blood clotting mechanism, and its function as a biochemical amplifier. Nature. 1964;202:498-499.
3. Asakai R, Chung DW, Davie EW, Seligsohn U. Factor XI deficiency in Ashkenazi Jews in Israel. N Engl J Med. 1991;325:153-158.
4. Gailani D, Renné T. The intrinsic pathway of coagulation: a target for treating thromboembolic disease? J Thromb Haemost. 2007;5:1106-1112.
5. Renné T, Gailani D. Role of Factor XII in hemostasis and thrombosis: clinical implications. Expert Rev Cardiovasc Ther. 2007;5:733-741.
6. Girolami A, Candeo N, De Marinis GB, Bonamigo E, Girolami B. Comparative incidence of thrombosis in reported cases of deficiencies of factors of the contact phase of blood coagulation. J Thromb Thrombolysis. 2011;31:57-63.
7. Rosen ED, Gailani D, Castellino FJ. FXI is essential for thrombus formation following FeCl3-induced injury of the carotid artery in the mouse. Thromb Haemost. 2002;87:774-776.
8. Cheng Q, Tucker EI, Pine MS, Sisler I, Matafonov A, Sun MF, White-Adams TC, Smith SA, Hanson SR, McCarty OJ, Renné T, Gruber A, Gailani D. A role for factor XIIa-mediated factor XI activation in thrombus formation in vivo. Blood. 2010;116:3981-3989.
9. Meijers JC, Tekelenburg WL, Bouma BN, Bertina RM, Rosendaal FR. High levels of coagulation factor XI as a risk factor for venous thrombosis. N Engl J Med. 2000;342:696-701.
10. Yang DT, Flanders MM, Kim H, Rodgers GM. Elevated factor XI activity levels are associated with an increased odds ratio for cerebrovascular events. Am J Clin Pathol. 2006;126:411-415.
11. Doggen CJ, Rosendaal FR, Meijers JC. Levels of intrinsic coagulation factors and the risk of myocardial infarction among men: opposite and synergistic effects of factors XI and XII. Blood. 2006;108: 4045-4051.
12. Salomon O, Steinberg DM, Koren-Morag N, Tanne D, Seligsohn U. Reduced incidence of ischemic stroke in patients with severe factor XI deficiency. Blood. 2008;111:4113-4117.
13. Salomon O, Apter S, Shaham D, Hiller N, Bar-Ziv J, Itzchak Y, Gitel S, Rosenberg N, Strauss S, Kaufman N, Seligsohn U. Risk factors associated with postpartum ovarian vein thrombosis. Thromb Haemost. 1999;82:1015-1019.
14. Salomon O, Steinberg DM, Zucker M, Varon D, Zivelin A, Seligsohn U. Patients with severe factor XI deficiency have a reduced incidence of deepvein thrombosis. Thromb Haemost. 2011;105:269-273.
15. Tucker EI, Marzec UM, White TC, Hurst S, Rugonyi S, McCarty OJ, Gailani D, Gruber A, Hanson SR. Prevention of vascular graft occlusion and thrombus-associated thrombin generation by inhibition of factor XI. Blood. 2009;113:936-944.
16. Gruber A, Hanson SR. Factor XI-dependence of surface-and tissue factorinitiated thrombus propagation in primates. Blood. 2003;102:953-955.
17. Wang X, Cheng Q, Xu L, Feuerstein GZ, Hsu MY, Smith PL, Seiffert DA, Schumacher WA, Ogletree ML, Gailani D. Effects of factor IX or factor XI deficiency on ferric chloride-induced carotid artery occlusion in mice. J Thromb Haemost. 2005;3:695-702.
18. Wang X, Smith PL, Hsu MY, Gailani D, Schumacher WA, Ogletree ML, Seiffert DA. Effects of factor XI deficiency on ferric chlorideinduced vena cava thrombosis in mice. J Thromb Haemost. 2006;4: 1982-1988.
19. Yamashita A, Nishihira K, Kitazawa T, Yoshihashi K, Soeda T, Esaki K, Imamura T, Hattori K, Asada Y. Factor XI contributes to thrombus propagation on injured neointima of the rabbit iliac artery. J Thromb Haemost. 2006;4:1496-1501.
20. Zhang H, Löwenberg EC, Crosby JR, MacLeod AR, Zhao C, Gao D, Black C, Revenko AS, Meijers JC, Stroes ES, Levi M, Monia BP. Inhibition of the intrinsic coagulation pathway factor XI by antisense oligonucleotides: a novel antithrombotic strategy with lowered bleeding risk. Blood. 2010;116:4684-4692.
21. Que Liu ED, Claudette B, Shuting X, John G, Brett P.M, Richard G, Sanjay B. Isis-fxirx, a novel and specific antisense inhibitor of factor xi, significantly reduces fxi antigen and activity and increases aptt without causing bleeding in healthy volunteers. Blood. 2011;118:209
22. Younis HS, Crosby J, Huh JI, Lee HS, Rime S, Monia B, Henry SP. Antisense inhibition of coagulation factor XI prolongs APTT without increased bleeding risk in cynomolgus monkeys. Blood. 2012;119:2401-2408.
23. Runge MS, Harker LA, Bode C, Ruef J, Kelly AB, Marzec UM, Allen E, Caban R, Shaw SY, Haber E, Hanson SR. Enhanced thrombolytic and antithrombotic potency of a fibrin-targeted plasminogen activator in baboons. Circulation. 1996;94:1412-1422.
24. Stasko J, Hudecek J, Kubisz P. [Thrombin activatable fibrinolysis inhibitor (TAFI) and its importance in the regulation of fibrinolysis]. Vnitr Lek. 2004;50:36-44.
25. Gruber A, Marzec UM, Bush L, Di Cera E, Fernández JA, Berny MA, Tucker EI, McCarty OJ, Griffin JH, Hanson SR. Relative antithrombotic and antihemostatic effects of protein C activator versus low-molecularweight heparin in primates. Blood. 2007;109:3733-3740.
26. Geary RS, Watanabe TA, Truong L, Freier S, Lesnik EA, Sioufi NB, Sasmor H, Manoharan M, Levin AA. Pharmacokinetic properties of 2'-O-(2-methoxyethyl)- modified oligonucleotide analogs in rats. J Pharmacol Exp Ther. 2001;296:890-897.
27. Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol. 2010;50:259-293.
28. Kastelein JJ, Wedel MK, Baker BF, Su J, Bradley JD, Yu RZ, Chuang E, Graham MJ, Crooke RM. Potent reduction of apolipoprotein B and lowdensity lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation. 2006;114:1729-1735.
29. Butler M, McKay RA, Popoff IJ, Gaarde WA, Witchell D, Murray SF, Dean NM, Bhanot S, Monia BP. Specific inhibition of PTEN expression reverses hyperglycemia in diabetic mice. Diabetes. 2002;51:1028-1034.
30. Richard S. Geary RZY, Andrew S, Arthur AL. Pharmacokinetic/ pharmacodynamic properties of phosphorothioate 2-o-(2-methoxyethyl)-modified antisense oligonucleotides in animals and man. Antisense Drug Technology, 2nd ed. Boca Raton, FL: Taylor & Francis Group. 2008:305-326
31. Tucker EI, Marzec UM, Berny MA, Hurst S, Bunting S, McCarty OJ, Gruber A, Hanson SR. Safety and antithrombotic efficacy of moderate platelet count reduction by thrombopoietin inhibition in primates. Sci Transl Med. 2010;2:37ra45.