Formulation development of lyophilized, long-term stable siRNA/oligoaminoamide polyplexes

European Journal of Pharmaceutics and Biopharmaceutics

Volumn 85, Issue 2, 2013, Pages 294-305

  Kasper, Julia Christina ^a   Troiber, Christina ^a   Küchler, Sarah ^a   Wagner, Ernst ^a   Friess, Wolfgang ^a  

^a UNIVERSITY OF MUNICH   (Germany)

Author keywords

Freeze drying; Lyophilization; Non viral RNA delivery; Polyplexes; siRNA delivery; Stability testing

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; AMIDE; DISULFIDE; EXCIPIENT; LACTOSUCROSE; SMALL INTERFERING RNA; SMALL INTERFERING RNA OLIGOAMINOAMIDE; SUCROSE; TREHALOSE; UNCLASSIFIED DRUG; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; BETA CYCLODEXTRIN DERIVATIVE;

ANIMAL CELL; ARTICLE; CONCENTRATION (PARAMETERS); CONTROLLED STUDY; CRYOPROTECTION; CYTOTOXICITY; DRUG FORMULATION; DRUG STABILITY; FREEZE DRYING; FREEZE THAWING; GENE SILENCING; GLASS TRANSITION TEMPERATURE; LYOPHILISATE; MOISTURE; MORPHOLOGY; MOUSE; NONHUMAN; OSMOLARITY; PARTICLE SIZE; PHOTON CORRELATION SPECTROSCOPY; TURBIDITY; X RAY POWDER DIFFRACTION; ANIMAL; CELL CULTURE; CHEMISTRY; FREEZING; MEDICINAL CHEMISTRY; PROCEDURES; TRANSITION TEMPERATURE; FREEZE-DRYING; METHODOLOGY; NON-VIRAL RNA DELIVERY; POLYPLEXES; SIRNA DELIVERY; STABILITY TESTING;

ANIMALS; BETA-CYCLODEXTRINS; CELLS, CULTURED; CHEMISTRY, PHARMACEUTICAL; DRUG STABILITY; EXCIPIENTS; FREEZE DRYING; FREEZING; MICE; PARTICLE SIZE; RNA, SMALL INTERFERING; TRANSITION TEMPERATURE;

FREEZE-DRYING; LYOPHILIZATION; NON-VIRAL RNA DELIVERY; POLYPLEXES; SIRNA DELIVERY; STABILITY TESTING;

EID: 84899780019     PISSN: 09396411     EISSN: 18733441     Source Type: Journal    
DOI: 10.1016/j.ejpb.2013.05.010     Document Type: Article

References (61)

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. M.L. Edelstein, M.R. Abedi, and J. Wixon Gene therapy clinical trials worldwide to 2007 - an update J. Gene Med. 9 2007 833 842
3. R. Almeida, and R.C. Allshire RNA silencing and genome regulation Trends Cell Biol. 15 2005 251 258
4. D.J. Gary, N. Puri, and Y.-Y. Won Polymer-based siRNA delivery: perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery J. Controlled Release 121 2007 64 73
5. P.D. Zamore, T. Tuschl, P.A. Sharp, and D.P. Bartel RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals Cell 101 2000 25 33
6. L. Aagaard, and J.J. Rossi RNAi therapeutics: principles, prospects and challenges Adv. Drug Deliv. Rev. 59 2007 75 86
7. L. Aagaard, and J.J. Rossi RNAi therapeutics: principles, prospects and challenges Adv. Drug Deliv. Rev. 59 2007 75 86
8. J. Wang, Z. Lu, M. Wientjes, and J. Au Delivery of siRNA therapeutics: barriers and carriers AAPS J. 12 2010 492 503
9. W. Kim, and S. Kim Efficient siRNA delivery with non-viral polymeric vehicles Pharm. Res. 26 2009 657 666
10. D. Schaffert, and E. Wagner Gene therapy progress and prospects: synthetic polymer-based systems Gene Therapy 15 2008 1131 1138
11. D.W. Pack, A.S. Hoffman, S. Pun, and P.S. Stayton Design and development of polymers for gene delivery Nat. Rev. Drug Discov. 4 2005 581 593
12. M. Meyer, and E. Wagner Recent developments in the application of plasmid DNA-based vectors and small interfering RNA therapeutics for cancer Hum. Gene Ther. 17 2006 1062 1076
13. A. Kwok, and S.L. Hart Comparative structural and functional studies of nanoparticle formulations for DNA and siRNA delivery Nanomed. Nanotechnol. Biol. Med. 7 2011 210 219
14. C. Troiber, and E. Wagner Nucleic acid carriers based on precise polymer conjugates Bioconjugate Chem. 22 2011 1737 1752
15. E. Wagner Polymers for siRNA delivery. Inspired by viruses to be targeted, dynamic, and precise Acc. Chem. Res. 45 2011 1005 1013
16. D. Schaffert, C. Troiber, E.E. Salcher, T. Fröhlich, I. Martin, N. Badgujar, C. Dohmen, D. Edinger, R. Kläger, G. Maiwald, K. Farkasova, S. Seeber, K. Jahn-Hofmann, P. Hadwiger, and E. Wagner Solid-phase synthesis of sequence-defined T-, i-, and U-shape polymers for pDNA and siRNA delivery Angew. Chem. Int. Ed. 50 2011 8986 8989
17. T.J. Anchordoquy, and G.S. Koe Physical stability of nonviral plasmid-based therapeutics J. Pharm. Sci. 89 2000 289 296
18. 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
19. M. Ogris, P. Steinlein, M. Kursa, K. Mechtler, R. Kircheis, and E. Wagner The size of DNA/transferrin-PEI complexes is an important factor for gene expression in cultured cells Gene Therapy 5 1998 1425 1433
20. 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
21. 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
22. 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
23. 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
24. 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
25. 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
26. J.C. Kasper, D. Schaffert, M. Ogris, E. Wagner, and W. Friess Development of a lyophilized plasmid/LPEI polyplex formulation with long-term stability - a step closer from promising technology to application J. Controlled Release 151 2011 246 255
27. C. Brus, E. Kleemann, A. Aigner, F. Czubayko, and T. Kissel Stabilization of oligonucleotide-polyethylenimine complexes by freeze-drying: physicochemical and biological characterization J. Controlled Release 95 2004 119 131
28. J.C. Zillies, K. Zwiorek, F. Hoffmann, A. Vollmar, T.J. Anchordoquy, G. Winter, and C. Coester Formulation development of freeze-dried oligonucleotide-loaded gelatin nanoparticles Eur. J. Pharm. Biopharm. 70 2008 514 521
29. W. Abdelwahed, G. Degobert, S. Stainmesse, and H. Fessi Freeze-drying of nanoparticles: formulation, process and storage considerations Adv. Drug Deliv. Rev. 58 2006 1688 1713
30. S. Wu, L. Putral, M. Liang, H.-I. Chang, N. Davies, and N. McMillan Development of a novel method for formulating stable siRNA-loaded lipid particles for in-vivo use Pharm. Res. 26 2009 512 522
31. A.K. Kundu, P.K. Chandra, S. Hazari, G. Ledet, Y.V. Pramar, S. Dash, and T.K. Mandal Stability of lyophilized siRNA nanosome formulations Int. J. Pharm. 423 2012 525 534
32. M.∅. Andersen, K.A. Howard, S.R. Paludan, F. Besenbacher, and J. Kjems Delivery of siRNA from lyophilized polymeric surfaces Biomaterials 29 2008 506 512
33. P. Yadava, M. Gibbs, C. Castro, and J. Hughes Effect of lyophilization and freeze-thawing on the stability of siRNA-liposome complexes AAPS PharmSciTech 9 2008 335 341
34. S. Werth, B. Urban-Klein, L. Dai, S. Höbel, M. Grzelinski, U. Bakowsky, F. Czubayko, and A. Aigner A low molecular weight fraction of polyethylenimine (PEI) displays increased transfection efficiency of DNA and siRNA in fresh or lyophilized complexes J. Controlled Release 112 2006 257 270
35. 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
36. T. Fröhlich, D. Edinger, R. Kläger, C. Troiber, E. Salcher, N. Badgujar, I. Martin, D. Schaffert, A. Cengizerogly, P. Hadwiger, H.-P. Vornlocher, and E. Wagner Structure-activity relationships of siRNA carriers based on sequence-defined oligo (ethane amino) amides J. Controlled Release 160 2012 532 541
37. C. Troiber, D. Edinger, P. Kos, L. Schreiner, R. Kläger, A. Herrmann, and E. Wagner Stabilizing effect of tyrosine trimers on pDNA and siRNA polyplexes Biomaterials 34 2013 1624 1633
38. C. Troiber, J.C. Kasper, S. Milani, M. Scheible, I. Martin, F. Schaubhut, S. Küchler, J. Rädler, F.C. Simmel, W. Friess, and E. Wagner Comparison of four different particle sizing methods for siRNA polyplex characterization Eur. J. Pharm. Biopharm. 84 2013 255 264
39. 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
40. F. Franks Freeze-drying of bioproducts: putting principles into practice Eur. J. Pharm. Biopharm. 45 1998 221 229
41. X. Tang, and M. Pikal Design of freeze-drying processes for pharmaceuticals: practical advice Pharm. Res. 21 2004 191 200
42. C. Körber, G. Rau, M.D. Cosman, and E.G. Cravalho Interaction of particles and a moving ice-liquid interface J. Cryst. Growth 72 1985 649 662
43. 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 β-galactosidase Arch. Biochem. Biophys. 384 2000 398 406
44. 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
45. 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
46. S. Höbel, R. Prinz, A. Malek, B. Urban-Klein, J. Sitterberg, U. Bakowsky, F. Czubayko, and A. Aigner Polyethylenimine PEI F25-LMW allows the long-term storage of frozen complexes as fully active reagents in siRNA-mediated gene targeting and DNA delivery Eur. J. Pharm. Biopharm. 70 2008 29 41
47. C.A. Hunter, and J.K.M. Sanders The nature of pi-pi interactions J. Am. Chem. Soc. 112 1990 5525 5534
48. R. Chelli, F.L. Gervasio, P. Procacci, and V. Schettino Stacking and T-shape competition in aromatic-aromatic amino acid interactions J. Am. Chem. Soc. 124 2002 6133 6143
49. J. Liu Physical characterization of pharmaceutical formulations in frozen and freeze-dried solid states: techniques and applications in freeze-drying development Pharm. Dev. Technol. 11 2006 3 28
50. 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
51. D.S. Allison, and T.J. Anchordoquy Mechanisms of protection of cationic lipid-DNA complexes during lyophilization J. Pharm. Sci. 89 2000 682 691
52. 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
53. J.C. Kasper, M.J. Pikal, and W. Friess Investigations on polyplex stability during the freezing step of lyophilization using controlled ice nucleation - the importance of residence time in the low viscosity fluid state J. Pharm. Sci. 102 2013 929 946
54. 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. Controlled Release 103 2005 465 479
55. J.G. Duguid, C. Li, M. Shi, M.J. Logan, H. Alila, A. Rolland, E. Tomlinson, J.T. Sparrow, and L.C. Smith A physicochemical approach for predicting the effectiveness of peptide-based gene delivery systems for use in plasmid-based gene therapy Biophys. J. 74 1998 2802 2814
56. E.C. Cho, Q. Zhang, and Y. Xia The effect of sedimentation and diffusion on cellular uptake of gold nanoparticles Nat. Nanotechnol. 6 2011 385 391
57. Ph.Eur., 2.9.19. Particulate contamination: sub-visible particles, in: European Pharmacopoeia 2008, pp. 300-302.
58. USP, (788), Particulate matter in injections, in: United States Pharmacopeia, 2010.
59. 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
60. B.C. Hancock, and G. Zografi The relationship between the glass transition temperature and the water content of amorphous pharmaceutical solids Pharm. Res. 11 1994 471 477
61. J. Yu, and T.J. Anchordoquy Effects of moisture content on the storage stability of dried lipoplex formulations J. Pharm. Sci. 98 2009 3278 3289