Enhancement of therapeutic protein in vivo activities through glycoengineering

Nature Biotechnology

Volumn 21, Issue 4, 2003, Pages 414-421

  Elliott, Steve ^a   Lorenzini, Tony ^a   Asher, Sheilah ^a   Aoki, Ken ^a   Brankow, David ^a   Buck, Lynette ^a   Busse, Leigh ^a   Chang, David ^a   Fuller, Janis ^a   Grant, James ^a   Hernday, Natasha ^a   Hokum, Martha ^a   Hu, Sylvia ^a   Knudten, Andrew ^a   Levin, Nancy ^a   Komorowski, Renee ^a   Martin, Frank ^a   Navarro, Rachell ^a   Osslund, Timothy ^a   Rogers, Gary ^a   Rogers, Norma ^a   Trail, Geri ^a   Egrie, Joan ^a   more..

^a AMGEN INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOTECHNOLOGY; CARBOHYDRATES; PATIENT TREATMENT;

THERAPEUTIC PROTEINS;

PROTEINS;

CARBOHYDRATE; LEPTIN; LIGAND; MPL LIGAND; NOVEL ERYTHROPOIESIS STIMULATING PROTEIN; PROTEIN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONSENSUS SEQUENCE; CONTROLLED STUDY; DRUG ACTIVITY; DRUG EFFICACY; FEMALE; GLYCOENGINEERING; MOLECULE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN ANALYSIS; PROTEIN ENGINEERING; PROTEIN GLYCOSYLATION; SCREENING; SEQUENCE ANALYSIS;

ANEMIA; ANIMALS; CHO CELLS; COS CELLS; CRICETINAE; DRUG DELIVERY SYSTEMS; ERYTHROPOIETIN, RECOMBINANT; EXCIPIENTS; FEMALE; GENETIC ENHANCEMENT; GLYCOPROTEINS; HUMANS; LEPTIN; MICE; MICE, INBRED BALB C; PROTEIN ENGINEERING; PROTEINS; RECOMBINANT PROTEINS; THROMBOPOIETIN;

ANIMALIA;

EID: 12244272391     PISSN: 10870156     EISSN: None     Source Type: Journal    
DOI: 10.1038/nbt799     Document Type: Article

References (44)

1. Delgado, C., Francis, G.E. & Fisher, D. The uses and properties of PEG-linked proteins. Crit. Rev Ther. Drug Carrier Systems 9, 249-304 (1992).
2. Sytkowski, A.J., Lunn, E.D., Risinger, M.A. & Davis, K.L. An erythropoietin fusion protein comprised of identical repeating domains exhibits enhanced biological properties. J. Biol Chem. 274, 24773-24778 (1999).
3. Sytkowski, A.J., Lunn, E.D., Davis, K.L., Feldman, L. & Siekman. S. Human erythropoietin dimers with markedly enhanced in vivo activity. Proc. Natl. Acad. Sci. USA 95, 1184-1188 (1998).
4. Dalle, B. et al. Dimeric erythropoietin fusion protein with enhanced erythropoietic activity in vitro and in vivo. Blood 97, 3776-3782 (2001).
5. Furuhashi, M. et al. Fusing the carboxy-terminal peptide of the chorionic gonadotropin (CG) β-subunit to the common α-subunit: retention of O-linked glycosylation and enhanced in vivo bioactivity of chimeric human CG. Mol. Endocrinol. 9, 54-63 (1995).
6. Kuai, L. et al. Plasminogen activator inhibitor-1 fused with erythropoietin (EPO) mimetic peptide (EMP) enhances the EPO activity of EMR J. Peptide Res. 56, 59-62(2000).
7. Marques, J.A., George, J.K., Smith, I.J., Bhakta, V. & Sheffield, W.P. A barbourin-albumin fusion protein that is slowly cleared in vivo retains the ability to inhibit platelet aggregation in vitro. Thromb. Haemost. 86, 902-908 (2001).
8. Sheffield, W.P., Smith, I.J., Syed, S. & Bhakta, V. Prolonged in vivo anticoagulant activity of a hirudin-albumin fusion protein secreted from Pichia pastoris. Blood Coag. Fibrinolysis 12, 433-443 (2001).
9. Moreland, L.W. Soluble tumor necrosis factor receptor (p75) fusion protein (ENBREL) as a therapy for rheumatoid arthritis. Rheum. Dis. Clin. North Am. 24, 579-591(1998).
10. Francis, G.E. et al. PEGylation of cytokines and other therapeutic proteins and peptides: the importance of biological optimisation of coupling techniques, Int. J. Hematol. 68, 1-18 (1998).
11. Coscarella, A. et al. The rhGM-CSF-EPO hybrid protein MEN 11300 induces anti-EPO antibodies and severe anaemia in rhesus monkeys. Cytokine 10, 964-969 (1998).
12. Gribben, J.G, et al. Development of antibodies to unprotected glycosylation sites on recombinant human GM-CSF. Lancet 335, 434-437 (1990).
13. Bause, E. Structural requirements of N-glycosylation of proteins. Studies with proline peptides as conformational probes. Biochem. J. 209, 331-336 (1983).
14. Roitsch, T. & Lehle, L. Structural requirements for protein N-glycosylation. Influence of acceptor peptides on cotranslational glycosylation of yeast invertase and site-directed mutagenesis around a sequon sequence. Eur. J. Biochem. 181, 525-529 (1989).
15. Imperiali, B. & Shannon, K.L. Differences between Asn-Xaa-Thr-containing peptides: a comparison of solution conformation and substrate behavior with oligosaccharyltransferase. Biochemistry 30, 4374-4380 (1991).
16. Browne, J.K. et al. Erythropoietin: gene cloning, protein structure, and biological properties. Cold Spring Harb. Symp. Quant. Biol. 51, 693-702 (1986).
17. Lin, F.K. et al. Cloning and expression of the human erythropoietin gene. Proc. Natl. Acad. Sci. USA 82, 7580-7584 (1985).
18. Egrie, J.C., Browne, J., Lai, P. & Lin, F.K. Characterization of recombinant monkey and human erythropoietin. Prog. Clin. Biol. Res. 191, 339-350 (1985).
19. Bartley, T.D. et al. Identification and cloning of a megakaryocyte growth and development factor that is a ligand for the cytokine receptor Mpl. Cell 77, 1117-1124 (1994).
20. Hunt, P. et al. Purification and biologic characterization of plasma-derived megakaryocyte growth and development factor. Blood 86, 540-547 (1995).
21. Zhang, Y. et al. Positional cloning of the mouse obese gene and its human homologue. Nature 372, 425-432 (1994).
22. Syed, R.S. et al. Efficiency of signalling through cytokine receptors depends critically on receptor orientation. Nature 395, 511-516 (1998).
23. Cheetham, J.C. et al. NMR structure of human erythropoietin and a comparison with its receptor-bound conformation. Nat. Struct. Biol. 5, 861-866 (1998).
24. Elliott, S., Lorenzini, T., Chang, D., Barzilay, J. & Delorme, E. Mapping of the active site of recombinant human erythropoietin. Blood 89, 493-502 (1997).
25. Elliott, S. et al. Fine-structure epitope mapping of anti-erythropoietin monoclonal antibodies reveals a model of recombinant human erythropoietin structure. Blood 87, 2702-2713 (1996),
26. Hirani, S., Bernasconi, R.J. & Rasmussen, J.R. Use of N-glycanase to release asparagine-linked oligosaccharides for structural analysis. Anal. Biochem. 162. 485-492 (1987).
27. Elliott, S. et al. Structural requirements for addition of O-linked carbohydrate to recombinant erythropoietin. Biochemistry 33, 11237-11245 (1994).
28. Elliott, S. et al. Isolation and characterization of conformation-sensitive anti-erythropoietin monoclonal antibodies: effect of disulfide bonds and carbohydrate on recombinant human erythropoietin structure. Blood 87, 2714-2722 (1996).
29. Egrie, J.C. & Browne, J.K. Development and characterization of novel erythropoiesis stimulating protein (NESP). Br. J. Cancer 84 (Suppl. 10), 3-10 (2001).
30. Macdougall, I.C. et al. Pharmacokinetics of novel erythropoiesis-stimulating protein compared with epoetin alfa in dialysis patients. J. Am. Soc. Nephrol. 10, 2392-2395(1999).
31. Heatherington, A.C., Schuller, J. & Mercer, A.J. Pharmacokinetics of novel erythropoiesis-stimulating protein (NESP) in cancer patients: preliminary report. Br. J. Cancer 84 (Suppl. 6), 11-16 (2001).
32. Locatelli, F., Baldamus, C.A., Villa, G., Ganea, A. & Martin de Francisco, A.L. Once-weekly compared with three-times-weekly subcutaneous epoetin β: results from a randomized, multicenter, therapeutic-equivalence study. Am. J. Kidney Dis. 40, 119-125 (2002).
33. Glaspy, J. et al, A dose-finding and safety study of novel erythropoiesis-stimulating protein (NESP) for the treatment of anaemia in patients receiving multicycle chemotherapy. Br. J. Cancer84 (Suppl. 23), 17-23 (2001).
34. Vansteenkiste, J. et al. Double-blind, placebo-controlled, randomized phase III trial of darbepoetin alfa in lung cancer patients receiving chemotherapy. J. Natl. Cancer Inst. 94, 1211-1220 (2002).
35. Nissenson, A.R. Novel erythropoiesis-stimulating protein for managing the anemia of chronic kidney disease. Am. J. Kidney Dis. 38, 1390-1397 (2001).
36. Macdougall, I.C. An overview of the efficacy and safety of novel erythropoiesis-stimulating protein (NESP). Nephrol. Dialysis Transplant. 16, 14-21 (2001).
37. Delorme, E. et al. Role of glycosylation on the secretion and biological activity of erythropoietin. Biochemistry 31, 9871-9876 (1992).
38. Wigler, M., Pellicer, A., Silverstein, S. & Axel, R. Biochemical transfer of single-copy eucaryotic genes using total cellular DNA as donor. Cell 14, 725-731 (1978).
39. Komatsu, N. et al. Establishment and characterization of an erythropoietin-dependent subline, UT-7/Epo, derived from human leukemia cell line, UT-7. Blood 82, 456-464 (1993).
40. Elliott, S., Lorenzini, T., Yanagihara, D., Chang, D. & Elliott, G. Activation of the erythropoietin (EPO) receptor by bivalent anti-EPO receptor antibodies. J. Biol. Chem. 271, 24691-24697 (1996).
41. Greenberger, J.S., Sakakeeny, M.A., Humphries, R.K., Eaves, C.J. & Eckner, R.J. Demonstration of permanent factor-dependent multipotential (erythroid/neutrophil/basophil) hematopoietic progenitor cell lines. Proc. Natl. Acad. Sci. USA 80, 2931-2935 (1983).
42. Tartaglia, L.A. et al. Identification and expression cloning of a leptin receptor, OBR. Cell 83, 1263-1271 (1995).
43. Jones, S.S., D'Andrea, A.D., Haines, L.L. & Wong, G.G. Human erythropoietin receptor: cloning, expression, and biologic characterization. Blood 76, 31-35 (1990).
44. Vigon, I. et al. Molecular cloning and characterization of MPL, the human homolog of the v-mpl oncogene: identification of a member of the hematopoietic growth factor receptor superfamily. Proc. Natl. Acad. Sci. USA 89, 5640-5644 (1992).