Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein

Journal of Thrombosis and Haemostasis

Volumn 11, Issue 1, 2013, Pages 132-141

  Peters, R T ^a   Toby, G ^a   Lu, Q ^a   Liu, T ^a   Kulman, J D ^a   Low, S C ^a   Bitonti, A J ^a   Pierce, G F ^a  

^a BIOGEN   (United States)

Author keywords

Factor VIII; Fc fusion; Hemophilia A; Long acting; RFVIIIFc

Indexed keywords

BLOOD CLOTTING FACTOR 8; HYBRID PROTEIN; IMMUNOGLOBULIN G1; MONOMER; PROTEINASE; THROMBIN; VON WILLEBRAND FACTOR;

ANIMAL EXPERIMENT; ARTICLE; ASSAY; BLOOD; BLOOD CLOTTING; EX VIVO STUDY; HEMOPHILIA A; HUMAN; HUMAN CELL; IN VITRO STUDY; MASS SPECTROMETRY; MOUSE; NONHUMAN; PARTIAL THROMBOPLASTIN TIME; PRIORITY JOURNAL; PROTEIN PROCESSING;

EID: 84873043409     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/jth.12076     Document Type: Article

References (17)

1. Peyvandi F, Jayandharan G, Chandy M, Srivastava A, Nakaya SM, Johnson MJ, Thompson AR, Goodeve A, Garagiola I, Lavoretano S, Menegatti M, Palla R, Spreafico M, Tagliabue L, Asselta R, Duga S, Mannucci PM. Genetic diagnosis of haemophilia and other inherited bleeding disorders. Haemophilia 2006; 12(Suppl. 3): 82-9.
2. Vehar GA, Keyt B, Eaton D, Rodriguez H, O'Brien DP, Rotblat F, Oppermann H, Keck R, Wood WI, Harkins RN, Tuddenhan EG, Lawn RM, Capon DJ. Structure of human factor VIII. Nature 1984; 312: 337-42.
3. Jankowski MA, Patel H, Rouse JC, Marzilli LA, Weston SB, Sharpe PJ. Defining 'full-length' recombinant factor VIII: a comparative structural analysis. Haemophilia 2007; 13: 30-7.
4. Sandberg H, Almstedt A, Brandt J, Castro VM, Gray E, Holmquist L, Lewin M, Oswaldsson U, Mikaelsson M, Jankowski MA, Bond M, Scoble HA. Structural and functional characterization of B-domain deleted recombinant factor VIII. Semin Hematol 2001; 38: 4-12.
5. Toole JJ, Pittman DD, Orr EC, Murtha P, Wasley LC, Kaufman RJ. A large region (approximately equal to 95kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc Natl Acad Sci USA 1986; 83: 5939-42.
6. Dumont JA, Liu T, Low SC, Zhang X, Kamphaus G, Sakorafas P, Fraley C, Drager D, Reidy T, McCue J, Franck HWG, Merricks EP, Nichols TC, Bitonti AJ, Pierce GF, Jiang H. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood 2012; 119: 3024-30.
7. Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood 2012; 119: 3031-7.
8. Chang JY, Monroe DM, Stafford DW, Brinkhous KM, Roberts HR. Replacing the first epidermal growth factor-like domain of factor IX with that of factor VII enhances activity in vitro and in canine hemophilia B. J Clin Invest 1997; 100: 886-92.
9. Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 1995; 10: 119-21.
10. Thim L, Vandahl B, Karlsson J, Klausen NK, Pedersen J, Krogh TN, Kjalke M, Petersen JM, Johnsen LB, Bolt G, Nørby PL, Steenstrup TD. Purification and characterization of a new recombinant factor VIII (N8). Haemophilia 2010; 16: 349-59.
11. Bitonti AJ, Dumont JA, Low SC, Peters RT, Kropp KE, Palombella VJ, Stattel JM, Lu Y, Tan CA, Song JJ, Garcia AM, Simister NE, Spiekermann GM, Lencer WI, Blumberg RS. Pulmonary delivery of an erythropoietin Fc fusion protein in non-human primates through an immunoglobulin transport pathway. Proc Natl Acad Sci USA 2004; 101: 9763-8.
12. Peters RT, Low SC, Kamphaus GD, Dumont JA, Amari JV, Lu Q, Zarbis-Papastoitsis G, Reidy TJ, Merricks EP, Nichols TC, Bitonti AJ. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood 2010; 115: 2057-64.
13. Vallee S, Rakhe S, Reidy T, Walker S, Lu Q, Sakorafas P, Low S, Bitonti A. Pulmonary administration of interferon Beta-1a-Fc fusion protein in non-human primates using an immunoglobulin transport pathway. J Interferon Cytokine Res 2011; 32: 178-84.
14. Dumont JA, Low SC, Peters RT, Bitonti AJ. Monomeric Fc fusions: impact on pharmacokinetic and biological activity of protein therapeutics. BioDrugs 2006; 20: 151-60.
15. Mikaelsson M, Oswaldsson U, Sandberg H. Influence of phospholipids on the assessment of factor VIII activity. Haemophilia 1998; 4: 646-50.
16. Christiansen ML, Balling KW, Persson E, Hilden I, Bagger-Sørensen A, Sørensen BB, Viuff D, Segel S, Klausen NK, Ezban M, Lethagen S, Steenstrup TD, Kjalke M. Functional characteristics of N8, a new recombinant FVIII. Haemophilia 2010; 16: 878-87.
17. Lenting PJ, van Schooten CJ, Denis CV. Clearance mechanisms of von Willebrand factor and factor VIII. J Thromb Haemost 2007; 5: 1353-60.