Evaluation of exogenous siRNA addition as a metabolic engineering tool for modifying biopharmaceuticals

Biotechnology Progress

Volumn 29, Issue 2, 2013, Pages 415-424

  Tummala, Seshu ^a   Titus, Michael ^a   Wilson, Lee ^a   Wang, Chunhua ^a   Ciatto, Carlo ^a   Thill, Greg ^a   Foster, Donald ^a   Li, Chen ^b   Szabo, Zoltan ^b   Guttman, Andras ^b   Bettencourt, Brian ^a   Jayaraman, Muthuswamy ^a   Deroot, Jack ^a   Kocisko, David ^a   Pollard, Stuart ^a   Charisse, Klaus ^a   Kuchimanchi, Satya ^a   Hinkle, Greg ^a   Milstein, Stuart ^a   Meyers, Rachel ^a   Wu, Shiaw Lin ^b   Karger, Barry L ^b   Rossomando, Anthony ^a,c   more..

^a ALNYLAM PHARMACEUTICALS   (United States)

^b NORTHEASTERN UNIVERSITY   (United States)

^c Synageva BioPharma Corp   (United States)

Author keywords

ADCC; Bioprocessing; Carbohydrate; Fucose; RNA interference; Transient

Indexed keywords

ADCC; BIOPROCESSING; FUCOSE; FUNCTIONAL CHARACTERISTICS; METABOLIC ENGINEERING APPROACH; MONOCLONAL ANTIBODIES (MAB); RNA INTERFERENCE; SMALL INTERFERING RNA (SIRNA);

BIOREACTORS; BOTTLES; CARBOHYDRATES; CELL CULTURE; CELLS; GENE EXPRESSION; MONOCLONAL ANTIBODIES; RNA; TRANSIENTS;

METABOLIC ENGINEERING;

FUCOSYLTRANSFERASE; GLYCOPROTEIN 6 ALPHA L FUCOSYLTRANSFERASE; GLYCOPROTEIN 6-ALPHA-L-FUCOSYLTRANSFERASE; MONOCLONAL ANTIBODY; SMALL INTERFERING RNA;

ANIMAL; ANTIBODY DEPENDENT CELLULAR CYTOTOXICITY; ARTICLE; CELL LINE; EQUIPMENT; EVALUATION; GENE SILENCING; GENETICS; HUMAN; IMMUNOLOGY; METABOLIC ENGINEERING; METABOLISM; METHODOLOGY; PROTEIN PROCESSING;

ANIMALS; ANTIBODIES, MONOCLONAL; ANTIBODY-DEPENDENT CELL CYTOTOXICITY; CELL LINE; FUCOSYLTRANSFERASES; GENE SILENCING; HUMANS; METABOLIC ENGINEERING; PROTEIN PROCESSING, POST-TRANSLATIONAL; RNA, SMALL INTERFERING;

EID: 84875936344     PISSN: 87567938     EISSN: 15206033     Source Type: Journal    
DOI: 10.1002/btpr.1667     Document Type: Article

References (37)

1. Ledford H.'Biosimilar' drugs poised to penetrate market.Nature. 2010;468:18-19.
2. Langer ES.Trends in capacity utilization for therapeutic monoclonal antibody production.MAbs. 2009;1:151-156.
3. Mortensen R.Production of a heterozygous mutant cell line by homologous recombination (single knockout).Curr Protoc Neurosci. 2011; Chapter 4:Unit 4 30.
4. Meek S, Buehr M, Sutherland L, Thomson A, Mullins JJ, Smith AJ, Burdon T.Efficient gene targeting by homologous recombination in rat embryonic stem cells.PLoS One. 2010;5:e14225.
5. Mortensen R.Production of a heterozygous mutant cell line by homologous recombination (single knockout).Curr Protoc Mol Biol. 2008;Chapter 23:Unit 23 25.
6. Yamane-Ohnuki N, Kinoshita S, Inoue-Urakubo M, Kusunoki M, Iida S, Nakano R, Wakitani M, Niwa R, Sakurada M, Uchida K, Shitara K, Satoh M.Establishment of FUT8 knockout Chinese hamster ovary cells: an ideal host cell line for producing completely defucosylated antibodies with enhanced antibody-dependent cellular cytotoxicity.Biotechnol Bioeng. 2004;87:614-622.
7. Malphettes L, Freyvert Y, Chang J, Liu PQ, Chan E, Miller JC, Zhou Z, Nguyen T, Tsai C, Snowden AW, Collingwood TN, Gregory PD, Cost GJ.Highly efficient deletion of FUT8 in CHO cell lines using zinc-finger nucleases yields cells that produce completely nonfucosylated antibodies.Biotechnol Bioeng. 2010;106:774-783.
8. Carroll D.Genome engineering with zinc-finger nucleases.Genetics. 2011;188:773-782.
9. Deng SS, Wang YZ, Ma D.Zinc finger nucleases and their application.Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2010;27:162-165.
10. Kanda Y, Imai-Nishiya H, Kuni-Kamochi R, Mori K, Inoue M, Kitajima-Miyama K, Okazaki A, Iida S, Shitara K, Satoh M.Establishment of a GDP-mannose 4, 6-dehydratase (GMD) knockout host cell line: a new strategy for generating completely non-fucosylated recombinant therapeutics.J Biotechnol. 2007;130:300-310.
11. Wong DC, Wong KT, Nissom PM, Heng CK, Yap MG.Targeting early apoptotic genes in batch and fed-batch CHO cell cultures.Biotechnol Bioeng. 2006;95:350-361.
12. Lim SF, Chuan KH, Liu S, Loh SO, Chung BY, Ong CC, Song Z.RNAi suppression of Bax and Bak enhances viability in fed-batch cultures of CHO cells.Metab Eng. 2006;8:509-522.
13. van Vugt MA, Medema RH.Polo-like kinase-1: activity measurement and RNAi-mediated knockdown.Methods Mol Biol. 2005;296:355-369.
14. Townsend JA, Wright DA, Winfrey RJ, Fu F, Maeder ML, Joung JK, Voytas DF.High-frequency modification of plant genes using engineered zinc-finger nucleases.Nature. 2009;459:442-445.
15. Pruett-Miller SM, Reading DW, Porter SN, Porteus MH.Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels.PLoS Genet. 2009;5:e1000376.
16. Tijsterman M, Plasterk RH.Dicers at RISC; the mechanism of RNAi.Cell. 2004;117:1-3.
17. Imai-Nishiya H, Mori K, Inoue M, Wakitani M, Iida S, Shitara K, Satoh M.Double knockdown of alpha1, 6-fucosyltransferase (FUT8) and GDP-mannose 4, 6-dehydratase (GMD) in antibody-producing cells: a new strategy for generating fully non-fucosylated therapeutic antibodies with enhanced ADCC.BMC Biotechnol. 2007;7:13.
18. Shitara K.Potelligent antibodies as next generation therapeutic antibodies.Yakugaku Zasshi. 2009;129:3-9.
19. Wong AW, Baginski TK, Reilly DE.Enhancement of DNA uptake in FUT8-deleted CHO cells for transient production of afucosylated antibodies.Biotechnol Bioeng. 2010;106:751-763.
20. Mizushima T, Yagi H, Takemoto E, Shibata-Koyama M, Isoda Y, Iida S, Masuda K, Satoh M, Kato K.Structural basis for improved efficacy of therapeutic antibodies on defucosylation of their Fc glycans.Genes Cells. 2011;16:1071-1080.
21. Reff ME, Carner K, Chambers KS, Chinn PC, Leonard JE, Raab R, Newman RA, Hanna N, Anderson DR.Depletion of B cells in vivo by a chimeric mouse human monoclonal antibody to CD20.Blood. 1994;83:435-445.
22. Brezinsky SC, Chiang GG, Szilvasi A, Mohan S, Shapiro RI, MacLean A, Sisk W, Thill G.A simple method for enriching populations of transfected CHO cells for cells of higher specific productivity.J Immunol Methods. 2003;277:141-155.
23. Manoharan M, Rajeev KG, Jayaraman M, Butler D, Kapoor M, Kainthan RK. Compositions for Nucleic Acid Delivery. WO 2011071860, 2011.
24. Addepalli H, Meena, Peng CG, Wang G, Fan Y, Charisse K, Jayaprakash KN, Rajeev KG, Pandey RK, Lavine G, Zhang L, Jahn-Hofmann K, Hadwiger P, Manoharan M, Maier MA.Modulation of thermal stability can enhance the potency of siRNA.Nucleic Acids Res. 2010;38:7320-7331.
25. Szabo Z, Guttman A, Bones J, Karger BL.Rapid high-resolution characterization of functionally important monoclonal antibody N-glycans by capillary electrophoresis.Anal Chem. 2011;83:5329-5336.
26. Wang Y, Wu SL, Hancock WS.Approaches to the study of N-linked glycoproteins in human plasma using lectin affinity chromatography and nano-HPLC coupled to electrospray linear ion trap-Fourier transform mass spectrometry.Glycobiology. 2006;16:514-523.
27. Wu SL, Kim J, Hancock WS, Karger B.Extended range proteomic analysis (ERPA): a new and sensitive LC-MS platform for high sequence coverage of complex proteins with extensive post-translational modifications-comprehensive analysis of beta-casein and epidermal growth factor receptor (EGFR).J Proteome Res. 2005;4:1155-1170.
28. Dall'Ozzo S, Tartas S, Paintaud G, Cartron G, Colombat P, Bardos P, Watier H, Thibault G.Rituximab-dependent cytotoxicity by natural killer cells: influence of FCGR3A polymorphism on the concentration-effect relationship.Cancer Res. 2004;64:4664-4669.
29. Shahbazian D, Parsyan A, Petroulakis E, Topisirovic I, Martineau Y, Gibbs BF, Svitkin Y, Sonenberg N.Control of cell survival and proliferation by mammalian eukaryotic initiation factor 4B.Mol Cell Biol. 2010;30:1478-1485.
30. Niwa R, Sakurada M, Kobayashi Y, Uehara A, Matsushima K, Ueda R, Nakamura K, Shitara K.Enhanced natural killer cell binding and activation by low-fucose IgG1 antibody results in potent antibody-dependent cellular cytotoxicity induction at lower antigen density.Clin Cancer Res. 2005;11:2327-2336.
31. Mori K, Kuni-Kamochi R, Yamane-Ohnuki N, Wakitani M, Yamano K, Imai H, Kanda Y, Niwa R, Iida S, Uchida K, Shitara K, Satoh M.Engineering Chinese hamster ovary cells to maximize effector function of produced antibodies using FUT8 siRNA.Biotechnol Bioeng. 2004;88:901-908.
32. von Horsten HH, Ogorek C, Blanchard V, Demmler C, Giese C, Winkler K, Kaup M, Berger M, Jordan I, Sandig V.Production of non-fucosylated antibodies by co-expression of heterologous GDP-6-deoxy-D-lyxo-4-hexulose reductase.Glycobiology. 2010;20:1607-1618.
33. Olivier S, Jacoby M, Brillon C, Bouletreau S, Mollet T, Nerriere O, Angel A, Danet S, Souttou B, Guehenneux F, Gauthier L, Berthome M, Vie H, Beltraminelli N, Mehtali M.EB66 cell line, a duck embryonic stem cell-derived substrate for the industrial production of therapeutic monoclonal antibodies with enhanced ADCC activity.MAbs. 2010;2:405-415.
34. Kurai J, Chikumi H, Hashimoto K, Yamaguchi K, Yamasaki A, Sako T, Touge H, Makino H, Takata M, Miyata M, Nakamoto M, Burioka N, Shimizu E.Antibody-dependent cellular cytotoxicity mediated by cetuximab against lung cancer cell lines.Clin Cancer Res. 2007;13:1552-1561.
35. Chung CH, Mirakhur B, Chan E, Le QT, Berlin J, Morse M, Murphy BA, Satinover SM, Hosen J, Mauro D, Slebos RJ, Zhou Q, Gold D, Hatley T, Hicklin DJ, Platts-Mills TA.Cetuximab-induced anaphylaxis and IgE specific for galactose-alpha-1, 3-galactose.N Engl J Med. 2008;358:1109-1117.
36. Bosques CJ, Collins BE, Meador JW 3rd, Sarvaiya H, Murphy JL, Dellorusso G, Bulik DA, Hsu IH, Washburn N, Sipsey SF, Myette JR, Raman R, Shriver Z, Sasisekharan R, Venkataraman G.Chinese hamster ovary cells can produce galactose-alpha-1, 3-galactose antigens on proteins.Nat Biotechnol. 2010;28:1153-1156.
37. Ghaderi D, Taylor RE, Padler-Karavani V, Diaz S, Varki A.Implications of the presence of N-glycolylneuraminic acid in recombinant therapeutic glycoproteins.Nat Biotechnol. 2010;28:863-867.