Development of a lyophilized plasmid/LPEI polyplex formulation with long-term stability-A step closer from promising technology to application

Journal of Controlled Release

Volumn 151, Issue 3, 2011, Pages 246-255

  Kasper, Julia Christina ^a   Schaffert, David ^a   Ogris, Manfred ^a   Wagner, Ernst ^a   Friess, Wolfgang ^a  

^a UNIVERSITY OF MUNICH   (Germany)

Author keywords

Freeze thawing; Long term stability; Lyophilization; Non viral gene delivery; Preserved transfection efficiency

Indexed keywords

FREEZE-THAWING; LONG TERM STABILITY; LYOPHILIZATION; NON-VIRAL GENE DELIVERY; PRESERVED TRANSFECTION EFFICIENCY;

AGGLOMERATION; AMINO ACIDS; EFFICIENCY; GENE THERAPY; GENE TRANSFER; GLASS; GLUCOSE; METABOLISM; MOISTURE; MOISTURE DETERMINATION; STABILITY; SUGAR (SUCROSE); SUSPENSIONS (FLUIDS); TEMPERATURE; THAWING; TURBIDITY; ZETA POTENTIAL;

GLASS TRANSITION;

DEXTRAN; HISTIDINE; HYDROXYPROPYLBETADEX; LACTOSUCROSE; POLYETHYLENEIMINE; POLYMER; POLYPLEX; POVIDONE; SUCROSE; TREHALOSE; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; DRUG FORMULATION; DRUG STABILITY; FREEZE DRYING; FREEZING; GLASS TRANSITION TEMPERATURE; MOISTURE; MOUSE; NONHUMAN; PARTICLE SIZE; PLASMID; PRIORITY JOURNAL; TURBIDITY; ZETA POTENTIAL;

ANIMALS; CALORIMETRY, DIFFERENTIAL SCANNING; CELL CULTURE TECHNIQUES; CELL LINE, TUMOR; CHEMISTRY, PHARMACEUTICAL; DRUG CARRIERS; DRUG STABILITY; EXCIPIENTS; FREEZE DRYING; LUCIFERASES; MICE; PARTICLE SIZE; PILOT PROJECTS; PLASMIDS; POLYETHYLENEIMINE; SURFACE PROPERTIES; TECHNOLOGY, PHARMACEUTICAL; TIME FACTORS; TRANSFECTION; X-RAY DIFFRACTION;

EID: 79957454818     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2011.01.003     Document Type: Article

References (51)

1. A. Pathak, S. Patnaik, and K.C. Gupta Recent trends in non-viral vector-mediated gene delivery Biotechnol. J. 4 2009 1559 1572
2. T.J. Anchordoquy, and G.S. Koe Physical stability of nonviral plasmid-based therapeutics J. Pharm. Sci. 89 2000 289 296 (Pubitemid 30152430)
3. T.J. Anchordoquy, S.D. Allison, M.d.C. Molina, L.G. Girouard, and T.K. Carson Physical stabilization of DNA-based therapeutics Drug Discov. Today 6 2001 463 470 (Pubitemid 32427661)
4. D. Schaffert, and E. Wagner Gene therapy progress and prospects: synthetic polymer-based systems Gene Ther. 15 2008 1131 1138
5. J. Clement, K. Kiefer, A. Kimpfler, P. Garidel, and R. Peschka-Süss Large-scale production of lipoplexes with long shelf-life Eur. J. Pharm. Biopharm. 59 2005 35 43 (Pubitemid 39573434)
6. S.D. Allison, and T.J. Anchordoquy Lyophilization of nonviral gene delivery systems M.A. Findeis, Nonviral Vectors for Gene Therapy: Methods and Protocols 2001 Humana Press Inc. New York 225 252
7. H. Talsma, J.-Y. Cherng, H. Lehrmann, M. Kursa, M. Ogris, W.E. Hennink, M. Cotten, and E. Wagner Stabilization of gene delivery systems by freeze-drying Int. J. Pharm. 157 1997 233 238 (Pubitemid 27471175)
8. F. Franks Freeze-drying of bioproducts: putting principles into practice Eur. J. Pharm. Biopharm. 45 1998 221 229 (Pubitemid 28270810)
9. X. Tang, and M. Pikal Design of freeze-drying processes for pharmaceuticals: practical advice Pharm. Res. 21 2004 191 200 (Pubitemid 38221965)
10. C. Brus, E. Kleemann, A. Aigner, F. Czubayko, and T. Kissel Stabilization of oligonucleotide-polyethylenimine complexes by freeze-drying: physicochemical and biological characterization J. Control. Release 95 2004 119 131 (Pubitemid 38167686)
11. W. Abdelwahed, G. Degobert, S. Stainmesse, and H. Fessi Freeze-drying of nanoparticles: formulation, process and storage considerations Adv. Drug Delivery Rev. 58 2006 1688 1713 (Pubitemid 44827432)
12. B.S. Chang, B.S. Kendrick, and J.F. Carpenter Surface-induced denaturation of proteins during freezing and its inhibition by surfactants J. Pharm. Sci. 85 1996 1325 1330 (Pubitemid 27014188)
13. K.A. Pikal-Cleland, N. Rodríguez-Hornedo, G.L. Amidon, and J.F. Carpenter Protein denaturation during freezing and thawing in phosphate buffer systems: monomeric and tetrameric [beta]-galactosidase Arch. Biochem. Biophys. 384 2000 398 406 (Pubitemid 32012817)
14. J.Y. Cherng, P.v.d. Wetering, H. Talsma, D.J.A. Crommelin, and W.E. Hennink Stabilization of polymer-based gene delivery systems Int. J. Pharm. 183 1999 25 28 (Pubitemid 29248822)
15. T.J. Anchordoquy, J.F. Carpenter, and D.J. Kroll Maintenance of transfection rates and physical characterization of lipid/DNA complexes after freeze-drying and rehydration Arch. Biochem. Biophys. 348 1997 199 206 (Pubitemid 27514688)
16. J.F. Carpenter, and J.H. Crowe The mechanism of cryoprotection of proteins by solutes Cryobiology 25 1988 244 255
17. G. Strauss, P. Schurtenberger, and H. Hauser The interaction of saccharides with lipid bilayer vesicles: stabilization during freeze-thawing and freeze-drying Biochim. Biophys. Acta 858 1986 169 180
18. W.L.J. Hinrichs, F.A. Manceñido, N.N. Sanders, K. Braeckmans, S.C. De Smedt, J. Demeester, and H.W. Frijlink The choice of a suitable oligosaccharide to prevent aggregation of PEGylated nanoparticles during freeze thawing and freeze drying Int. J. Pharm. 311 2006 237 244
19. T.J. Anchordoquy, T.K. Armstrong, and M.d.C. Molina Low molecular weight dextrans stabilize nonviral vectors during lyophilization at low osmolalities: concentrating suspensions by rehydration to reduced volumes J. Pharm. Sci. 94 2005 1226 1236 (Pubitemid 40904187)
20. T.J. Anchordoquy, T.K. Armstrong, M.D.C. Molina, S.D. Allison, Y. Zhang, M.M. Patel, Y.K. Lentz, and G.S. Koe Formulation considerations for DNA-based therapeutics D.R. Lu, S. Oeie, Cellular Drug Delivery 2004 Humana Press Inc. Totowa 237 263
21. M.d.C. Molina, S.D. Allison, and T.J. Anchordoquy Maintenance of nonviral vector particle size during the freezing step of the lyophilization process is insufficient for preservation of activity: insight from other structural indicators J. Pharm. Sci. 90 2001 1445 1455 (Pubitemid 32979740)
22. S.D. Allison, and J.A. Thomas Mechanisms of protection of cationic lipid-DNA complexes during lyophilization J. Pharm. Sci. 89 2000 682 691
23. S.D. Allison, M.d.C. Molina, and T.J. Anchordoquy Stabilization of lipid/DNA complexes during the freezing step of the lyophilization process: the particle isolation hypothesis Biochim. Biophys. Acta 1468 2000 127 138
24. B. Li, S. Li, Y. Tan, D.B. Stolz, S.C. Watkins, L.H. Block, and L. Huang Lyophilization of cationic lipid-protamine-DNA (LPD) complexes J. Pharm. Sci. 89 2000 355 364 (Pubitemid 30152436)
25. O. Zelphati, C. Nguyen, M. Ferrari, J. Felgner, Y. Tsai, and P.L. Felgner Stable and monodisperse lipoplex formulations for gene delivery Gene Ther. 5 1998 1272 1282 (Pubitemid 28448437)
26. T.J. Anchordoquy, L.G. Girouard, J.F. Carpenter, and D.J. Kroll Stability of lipid/DNA complexes during agitation and freeze-thawing J. Pharm. Sci. 87 1998 1046 1051 (Pubitemid 28452102)
27. J.-Y. Cherng, H. Talsma, D. Crommelin, and W. Hennink Long term Stability of poly((2-dimethylamino)ethyl methacrylate)-based gene delivery systems Pharm. Res. 16 1999 1417 1423 (Pubitemid 29449380)
28. J.-Y. Cherng, P. van de Wetering, H. Talsma, D.J.A. Crommelin, and W.E. Hennink Freeze-drying of poly((2-dimethylamino)ethyl methacrylate)-based Gene delivery systems Pharm. Res. 14 1997 1838 1841 (Pubitemid 28062935)
29. T.K. Armstrong, and T.J. Anchordoquy Immobilization of nonviral vectors during the freezing step of lyophilization J. Pharm. Sci. 93 2004 2698 2709 (Pubitemid 39506620)
30. M. Kursa, G.F. Walker, V. Roessler, M. Ogris, W. Roedl, R. Kircheis, and E. Wagner Novel shielded transferrin-polyethylene glycol-polyethylenimine/DNA complexes for systemic tumor-targeted gene transfer Bioconjugate Chem. 14 2002 222 231 (Pubitemid 36169036)
31. M. Lenter, P. Garidel, J. Pelisek, E. Wagner, and M. Ogris Stabilized Nonviral Formulations for the Delivery of MCP-1 Gene into Cells of the Vasculoendothelial System Pharm. Res. 21 2004 683 691 (Pubitemid 38457292)
32. D. Schaffert, M. Kiss, W. Rödl, A. Shir, A. Levitzki, M. Ogris, E. Wagner, Poly(I:C) mediated tumor growth suppression in EGF-receptor overexpressing tumors using EGF-polyethylene glycol-linear polyethylenimine as carrier, Pharm. Res., doi:10.1007/s11095-010-0225-4.
33. J.C. Kasper, D. Schaffert, M. Ogris, E. Wagner, and W. Friess The establishment of an up-scaled micro-mixer method allows the standardized and reproducible preparation of well-defined plasmid/LPEI polyplexes Eur. J. Pharm. Biopharm. 77 2011 182 185
34. -2.9.19. Particulate contamination: sub-visible particles European Pharmacopoeia 2008 300 302
35. T. Mosmann Rapid colorimetric assay for cellular growth and survival: application to proliferation and cytotoxicity assays J. Immunol. Methods 65 1983 55 63 (Pubitemid 14203433)
36. http://www.accessdata.fda.gov/scripts/cder/iig/index.cfm, Inactive Ingredients in Approved FDA Drugs, 2009.
37. M. Ogris Gene delivery using polyethylenimine and copolymers M.M. Amiji, Polymeric Gene Delivery 2005 CRC Press LLC, Boca Raton Principles and Applications 97 106
38. E. Lai, and J.H. van Zanten Monitoring DNA/poly-l-lysine polyplex formation with time-resolved multiangle laser light scattering Biophys. J. 80 2001 864 873 (Pubitemid 32128337)
39. W.L.J. Hinrichs, N.N. Sanders, S.C. De Smedt, J. Demeester, and H.W. Frijlink Inulin is a promising cryo- and lyoprotectant for PEGylated lipoplexes J. Control. Release 103 2005 465 479 (Pubitemid 40342542)
40. J.H. Crowe, L.M. Crowe, and J.F. Carpenter Preserving dry biomaterials: the water replacement hypothesis. Part 1 BioPharm 6 1993 28 29 32-33
41. S.C. De Smedt, J. Demeester, and W.E. Hennink Cationic polymer based gene delivery systems Pharm. Res. 17 2000 113 126 (Pubitemid 30158358)
42. K.J. Hartauer, G.N. Arbuthnot, S.W. Baertschi, R.A. Johnson, W.D. Luke, N.G. Pearson, E.C. Rickard, C.A. Tingle, P.K.S. Tsang, and R.E. Wiens Influence of peroxide impurities in povidone and crospovidone on the stability of raloxifene hydrochloride in tablets: identification and control of an oxidative degradation product Pharm. Dev. Technol. 5 2000 303 310 (Pubitemid 30602837)
43. V. Kumar, and D. Kalonia Removal of peroxides in polyethylene glycols by vacuum drying: implications in the stability of biotech and pharmaceutical formulations AAPS PharmSciTech 7 2006 E47 E53
44. M. Messner, S.V. Kurkov, P. Jansook, and T. Loftsson Self-assembled cyclodextrin aggregates and nanoparticles Int. J. Pharm. 387 2010 199 208
45. <788> Particulate matter in injections USP (U.S. Pharmacopeia) 34 2011 326 328
46. T.G. Fox, and P.J. Flory Second-order transition temperatures and related properties of polystyrene. I. Influence of molecular weight J. Appl. Phys. 21 1950 581 591
47. W. Abdelwahed, G. Degobert, and H. Fessi Investigation of nanocapsules stabilization by amorphous excipients during freeze-drying and storage Eur. J. Pharm. Biopharm. 63 2006 87 94 (Pubitemid 43776204)
48. M.d.C. Molina, T.K. Armstrong, Y. Mayank, Z.M. Patel, Y.K. Lentz, and T.J. Anchordoquy The stability of lyophilized lipid/DNA complexes during prolonged storage J. Pharm. Sci. 93 2004 2259 2273 (Pubitemid 39129624)
49. R. Fuhrherr, Spray-dried antibody powders for pulmonary application., in, Ph. D. Thesis Ludwig-Maximilian University, Munich, 2005.
50. S.J. Prestrelski, K.A. Pikal, and T. Arakawa Optimization of lyophilization conditions for recombinant human interleukin-2 by dried-state conformational analysis using Fourier-transform infrared spectroscopy Pharm. Res. 12 1995 1250 1259
51. E.Y. Shalaev, and G. Zografi How does residual water affect the solid-state degradation of drugs in the amorphous state? J. Pharm. Sci. 85 1996 1137 1141 (Pubitemid 26372881)