Challenges in the pharmaceutical development of lipid-based short interfering ribonucleic acid therapeutics

Expert Opinion on Drug Delivery

Volumn 9, Issue 2, 2012, Pages 171-182

  Gindy, Marian E ^a   Leone, Anthony M ^a   Cunningham, James J ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

Author keywords

assembly; biocomparability; lipid; lipid analysis; nanoparticle; pharmaceutical; SiRNA; SiRNA analysis; therapeutic

Indexed keywords

ALN TTR; ALN VSP; APOLIPOPROTEIN B; ATU 027; LIPID; NANOPARTICLE; POLO LIKE KINASE 1; SMALL INTERFERING RNA; TKM APOLIPOPROTEIN B; TKM PLK1; UNCLASSIFIED DRUG; VASCULOTROPIN;

DRUG FORMULATION; DRUG PACKAGING; DRUG STABILITY; DRUG STORAGE; IN VIVO STUDY; PARTICLE SIZE; PHYSICAL CHEMISTRY; PRECIPITATION; REVIEW; RNA INTERFERENCE;

CHEMISTRY, PHARMACEUTICAL; DRUG DELIVERY SYSTEMS; DRUG DISCOVERY; DRUG STABILITY; HUMANS; LIPIDS; NANOPARTICLES; RNA INTERFERENCE; RNA, SMALL INTERFERING; TECHNOLOGY, PHARMACEUTICAL;

EID: 84856098210     PISSN: 17425247     EISSN: 17447593     Source Type: Journal    
DOI: 10.1517/17425247.2012.642363     Document Type: Review

References (93)

1. Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391(6669):806-11 (Pubitemid 28099681)
2. Dykxhoorn DM, Lieberman J. The silent revolution: RNA interference as basic biology, research tool, and therapeutic. Annu Rev Med 2005;56:401-23 (Pubitemid 40299790)
3. De Fougerolles A, Vornlocher H-P, Maraganore J, et al. Interfering with disease: a progress report on siRNA-based therapeutics. Nat Rev Drug Discov 2007;6(6):443-53 (Pubitemid 47064567)
4. Pecot CV, Calin GA, Coleman RL, et al. RNA interference in the clinic: challenges and future directions. Nat Rev Cancer 2011;11(1):59-67
5. Juliano R, Bauman J, Kang H, et al. Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides. Mol Pharm 2009;6(3):686-95
6. Karmali PP, Simberg D. Interactions of nanoparticles with plasma proteins: implication on clearance and toxicity of drug delivery systems. Expert Opin Drug Deliv 2011;8(3):343-57
7. Whitehead KA, Langer R, Anderson DG. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 2009;8(2):129-38
8. Fenske DB, Cullis PR. Liposomal nanomedicines. Expert Opin Drug Deliv 2008;5(1):25-44 (Pubitemid 351234108)
9. Rozema DB, Lewis DL, Wakefield DH, et al. Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc Natl Acad Sci USA 2007;104(32):12982-7 (Pubitemid 351737577)
10. Jeong JH, Mok H, Oh YK, et al. siRNA conjugate delivery systems. Bioconjug Chem 2009;20(1):5-14
11. Soutschek J, Akinc A, Bramlage B, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004;432(7014):173-8 (Pubitemid 39545841)
12. Davis ME, Pun SH, Bellocq NC, et al. Self-assembling nucleic acid delivery vehicles via linear, water-soluble, cyclodextrin-containing polymers. Curr Med Chem 2004;11(2):179-97 (Pubitemid 38128844)
13. Xu ZP, Niebert M, Porazik K, et al. Subcellular compartment targeting of layered double hydroxide nanoparticles. J Control Release 2008;130(1):86-94
14. Derfus AM, Chen AA, Min DH, et al. Targeted quantum dot conjugates for siRNA delivery. Bioconjug Chem 2007;18(5):1391-6 (Pubitemid 47464045)
15. Ming X. Cellular delivery of siRNA and antisense oligonucleotides via receptor-mediated endocytosis. Expert Opin Drug Deliv 2011;8(4):435-49
16. Felgner PL, Gadek TR, Holm M, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987;84(21):7413-17
17. Hafez IM, Maurer N, Cullis PR. On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids. Gene Ther 2001;8(15):1188-96 (Pubitemid 32776393)
18. Hirko A, Tang F, Hughes JA. Cationic lipid vectors for plasmid DNA delivery. Curr Med Chem 2003;10(14):1185-93 (Pubitemid 36763929)
19. Ming X, Sato K, Juliano RL. Unconventional internalization mechanisms underlying functional delivery of antisense oligonucleotides via cationic lipoplexes and polyplexes. J Control Release 2011;153(1):83-92
20. Lu JJ, Langer R, Chen J. A Novel Mechanism Is Involved in Cationic Lipid-Mediated Functional siRNA Delivery. Mol Pharm 2009;6(3):763-71
21. Hui SW, Langner M, Zhao YL, et al. The role of helper lipids in cationic liposome-mediated gene transfer. Biophys J 1996;71(2):590-9 (Pubitemid 26289143)
22. Kirby C, Clarke J, Gregoriadis G. Effect of the cholesterol content of small unilamellar liposomes on their stability in vivo and in vitro. Biochem J 1980;186(2):591-8
23. Koltover I, Salditt T, Radler JO, et al. An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. Science 1998;281(5373):78-81 (Pubitemid 28354046)
24. Radler JO, Koltover I, Salditt T, et al. Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science 1997;275(5301):810-14 (Pubitemid 27074877)
25. Zuhorn IS, Oberle V, Visser WH, et al. Phase behavior of cationic amphiphiles and their mixtures with helper lipid influences lipoplex shape, DNA translocation, and transfection efficiency. Biophys J 2002;83(4):2096-108
26. Simberg D, Danino D, Talmon Y, et al. Phase behavior, DNA ordering, and size instability of cationic lipoplexes - Relevance to optimal transfection activity. J Biol Chem 2001;276(50):47453-9
27. Simberg D, Weisman S, Talmon Y, et al. DOTAP (and other cationic lipids): Chemistry, biophysics, and transfection. Crit Rev Ther Drug Carrier Syst 2004;21(4):257-317 (Pubitemid 41647579)
28. Stanton MG, Colletti SL. Medicinal Chemistry of siRNA Delivery. J Med Chem 2010;53(22):7887-901
29. Ambegia E, Ansell S, Cullis P, et al. Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim Biophys Acta 2005;1669(2):155-63 (Pubitemid 40704733)
30. Romberg B, Hennink WE, Storm G. Sheddable coatings for long-circulating nanoparticles. Pharm Res 2008;25(1):55-71
31. Akinc A, Querbes W, De S, et al. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther 2010;18(7):1357-64
32. Gregoriadis G. editor. Liposome Technology. 3rd edition. Informa Healthcare; New York: 2010
33. Wheeler JJ, Palmer L, Ossanlou M, et al. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther 1999;6(2):271-81 (Pubitemid 29089926)
34. Stuart DD, Kao GY, Allen TM. A novel, long-circulating, and functional liposomal formulation of antisense oligodeoxynucleotides targeted against MDR1. Cancer Gene Ther 2000;7(3):466-75 (Pubitemid 30210414)
35. Wu SY, Putral LN, Liang MT, et al. Development of a novel method for formulating stable sirna-loaded lipid particles for in vivo use. Pharm Res 2009;26(3):512-22
36. Lurquin PF. Entrapment of Plasmid DNA by Liposomes and their interactions with Plant-Protoplasts. Nucleic Acids Res 1979;6(12):3773-84
37. Felgner PL, Gadek TR, Holm M, et al. Lipofection - a highly efficient, lipid-mediated dna-transfection procedure. Proc Natl Acad Sci USA 1987;84(21):7413-17
38. Maurer N, Wong KF, Stark H, et al. Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes. Biophys J 2001;80(5):2310-26 (Pubitemid 32401995)
39. Horn D, Schmidt HW, Ditter W, et al. et al. inventors. Preparation of finely divided pulverulent carotinoid and retinoid compositions patent. 4522743; 1985
40. Texter J, Travis WB, Flow V. inventors Microprecipitation process for dispersing photographic filter dyes patent. 5624467; 1997
41. Midler ME, Paul EL, Whittington EF, et al. inventors. Crystallization Method to Improve Crystal Structure and Size patent. 5314506; 1994
42. Violante MB, Fischer HW. inventors. Method for making uniformly-sized particles from insoluble compounds patent. 4997454; 1991
43. Johnson BK, Prud'homme RK. Flash NanoPrecipitation of organic actives and block copolymers using a confined impinging jets mixer. Aust J Chem 2003;56(10):1021-4
44. Gindy ME, Panagiotopoulos AZ, Prud'homme RK. Composite block copolymer stabilized nanoparticles: simultaneous encapsulation of organic actives and inorganic nanostructures. Langmuir 2008;24(1):83-90
45. Gindy ME, Prud'homme RK. Multifunctional nanoparticles for imaging, delivery and targeting in cancer therapy. Expert Opin Drug Deliv 2009;6(8):865-78
46. Jeffs LB, Palmer LR, Ambegia EG, et al. A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA. Pharm Res 2005;22(3):362-72 (Pubitemid 40558494)
47. Zimmermann TS, Lee ACH, Akinc A, et al. RNAi-mediated gene silencing in non-human primates. Nature 2006;441(7089):111-14
48. Nguyen NT, Wu ZG. Micromixers - a review. J Micromech Microeng 2005;15(2):R1-R16
49. Johnson TJ, Ross D, Locascio LE. Rapid microfluidic mixing. Anal Chem 2002;74(1):45-51 (Pubitemid 34044422)
50. Bothe D, Sternich C, Warnecke HJ. Fluid mixing in a T-shaped micro-mixer. Chem Eng Sci 2006;61(9):2950-8 (Pubitemid 43277398)
51. Mahajan AJ, Kirwan DJ. Micromixing effects in a two-impinging-jets precipitator. Aiche J 1996;42(7):1801-14
52. Marcant B, David R. Experimental-evidence for and prediction of micromixing effects in precipitation. Aiche J 1991;37(11):1698-710
53. Mullin JW. Crystallization. 3rd edition. Butterworth Heinemann; Oxford,UK: 1993
54. Garside J, Mersmann A, Nyvlt J. Measurements of Crystal Growth and Nucleation Rates. 2nd edition. Institution of Chemical Engineers; Rugby: 2002
55. Semple SC, Klimuk SK, Harasym TO, et al. Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures. Biochim Biophys Acta-Biomembr 2001;1510(1-2):152-66 (Pubitemid 32149893)
56. Holland JW, Hui C, Cullis PR, et al. Poly(ethylene glycol)-lipid conjugates regulate the calcium-induced fusion of liposomes composed of phosphatidylethanolamine and phosphatidylserine. Biochemistry 1996;35(8):2618-24
57. Johnsson M, Hansson P, Edwards K. Spherical micelles and other self-assembled structures in dilute aqueous mixtures of poly(ethylene glycol) lipids. J Phys Chem B 2001;105(35):8420-30 (Pubitemid 35338819)
58. Lasic DD, Needham D. The ''Stealth'' liposome: a prototypical biomaterial. Chem Rev 1995;95(8):2601-28
59. Webb MS, Saxon D, Wong FMP, et al. Comparison of different hydrophobic anchors conjugated to poly(ethylene glycol): effects on the pharmacokinetics of liposomal vincristine. Biochim Biophys Acta-Biomembr 1998;1372(2):272-82 (Pubitemid 28451550)
60. Ashok B, Arleth L, Hjelm RP, et al. In vitro characterization of PEGylated phospholipid micelles for improved drug solubilization: effects of PEG chain length and PC incorporation. J Pharm Sci 2004;93(10):2476-87 (Pubitemid 39319431)
61. Malmsten M, Emoto K, Van Alstine JM. Effect of chain density on inhibition of protein adsorption by poly(ethylene glycol) based coatings. J Colloid Interface Sci 1998;202(2):507-17 (Pubitemid 28394232)
62. Taylor P. Ostwald ripening in emulsions. Adv Colloid Interface Sci 1998;75(2):107-63 (Pubitemid 128413054)
63. Karmali PP, Simberg D. Interactions of nanoparticles with plasma proteins: implication on clearance and toxicity of drug delivery systems. Expert Opin Drug Deliv 2011;8(3):343-57
64. Kawakami S, Hashida M. Targeted delivery systems of small interfering RNA by systemic administration. Drug Metab Pharmacokinet 2007;22(3):142-51
65. Zhou L. Draft Guidance for Industry on Liposome Drug Products: Chemistry, Manufacturing, and Controls; Human Pharmacokinetics and Bioavailability; and Labeling Documentation 2002 08/21/02:15
66. Grit M, Crommelin JA. Chemical-stability of liposomes - implications for their physical stability. Chem Phys Lipids 1993;64(1-3):3-18 (Pubitemid 23290861)
67. Hovorka SW, Schoneich C. Oxidative degradation of pharmaceuticals: theory, mechanisms and inhibition. J Pharm Sci 2001;90(3):253-69 (Pubitemid 32209888)
68. Frankel EN. Recent advances in lipid oxidation. J Sci Food Agric 1991;54(4):495-511
69. Cosgrove JP, Church DF, Pryor WA. The Kinetics of the Autoxidation of Polyunsaturated Fatty-Acids. Lipids 1987;22(5):299-304
70. Zuidam NJ, Crommelin DJA. Chemical hydrolysis of phospholipids. J Pharm Sci 1995;84(9):1113-19
71. Grit M, Desmidt JH, Struijke A, et al. Hydrolysis of phosphatidylcholine in aqueous liposome dispersions. Int J Pharm 1989;50(1):1-6 (Pubitemid 19049338)
72. Crommelin DJA. Influence of lipid-composition and ionic-strength on the physical stability of liposomes. J Pharm Sci 1984;73(11):1559-63 (Pubitemid 15205531)
73. Grit M, Crommelin DJA. The effect of aging on the physical stability of liposome dispersions. Chem Phys Lipids 1992;62(2):113-22
74. Krotz AH, Mehta RC, Hardee GE. Peroxide-mediated desulfurization of phosphorothioate oligonucleotides and its prevention. J Pharm Sci 2005;94(2):341-52 (Pubitemid 40293330)
75. Crommelin DJA, Grit M, Talsma H, et al. Liposomes as carriers for drugs and antigens - approaches to preserve their long-term stability. Drug Dev Ind Pharm 1994;20(4):547-56 (Pubitemid 24115166)
76. Samuni AM, Lipman A, Barenholz Y. Damage to liposomal lipids: protection by antioxidants and cholesterol-mediated dehydration. Chem Phys Lipids 2000;105(2):121-34
77. Armengol X, Estelrich J. Physical stability of different liposome compositions obtained by extrusion method. J Microencapsul 1995;12(5):525-35
78. Beckwith ALJ, Eichinger PH, Mooney BA, et al. Amine Autoxidation in Aqueous-Solution. Aust J Chem 1983;36(4):719-39
79. Grit M, Crommelin DJA. The effect of surface-charge on the hydrolysis kinetics of partially hydrogenated egg phosphatidylcholine and egg phosphatidylglycerol in aqueous liposome dispersions. Biochim Biophys Acta 1993;1167(1):49-55 (Pubitemid 23083036)
80. Parasassi T, Giusti AM, Raimondi M, et al. Abrupt modifications of phospholipid-bilayer properties at critical cholesterol concentrations. Biophys J 1995;68(5):1895-902
81. Ausborn M, Nuhn P, Schreier H. Stabilization of liposomes by freeze-thaw and lyophilization techniques - problems and opportunities. Eur J Pharm Biopharm 1992;38(4):133-9
82. Guideline. ICH: comparability of biotechnological/biological products subject to changes in their manufacturing process Q5E. Fed Regist 2005;70(125):2
83. De Geest B, Jacobs F, Wisse E. The role of liver sinusoidal cells in hepatocyte-directed gene transfer. Am J Pathol 2010;176(1):14-21
84. De Geest B, Lievens J, Snoeys J, et al. The size of sinusoidal fenestrae is a critical determinant of hepatocyte transduction after adenoviral gene transfer. Gene Ther 2004;11(20):1523-31 (Pubitemid 39359460)
85. De Geest B, Snoeys J, Lievens J, et al. Species differences in transgene DNA uptake in hepatocytes after adenoviral transfer correlate with the size of endothelial fenestrae. Gene Ther 2007;14(7):604-12 (Pubitemid 46444534)
86. De Geest B, Wisse E, Jacobs F, et al. The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer. Gene Ther 2008;15(17):1193-9
87. Wisse E, Braet F, Luo Dz, et al. Structure and function of sinusoidal lining cells in the liver. Toxicol Pathol 1996;24(1):100-11 (Pubitemid 26066425)
88. Kiwada H, Ishida T, Atobe K, et al. Accelerated blood clearance of PEGylated liposomes upon repeated injections: effect of doxorubicin- encapsulation and high-dose first injection. J Control Release 2006;115(3):251-8 (Pubitemid 44634849)
89. Fumoto S, Kawakami S, Shigeta K, et al. Interaction with blood components plays a crucial role in asialoglycoprotein receptor-mediated in vivo gene transfer by galactosylated lipoplex. J Pharmacol Exp Ther 2005;315(2):484-93 (Pubitemid 41500711)
90. Hrynyk R, Storm G, Metselaar B, et al. Pharmacokinetics of liposomes designed to carry glucocorticoids. Pol J Pharmacol 2003;55(6):1063-70 (Pubitemid 38128943)
91. Senior J, Delgado C, Fisher D, et al. Influence of surface hydrophilicity of liposomes on their interaction with plasma protein and clearance from the circulation: studies with poly(ethylene glycol)-coated vesicles. Biochim Biophys Acta 1991;1062(1):77-82
92. Hafez IM, Cullis PR. Roles of lipid polymorphism in intracellular delivery. Adv Drug Deliv Rev 2001;47(2-3):139-48 (Pubitemid 32324111)
93. Mach H, Volkin DB, Troutman RD, et al. Disassembly and reassembly of yeast-derived recombinant human papillomavirus virus-like particles (HPV VLPs). J Pharm Sci 2006;95(10):2195-206 (Pubitemid 44568530)