Multidimensional methods for the formulation of biopharmaceuticals and vaccines

Journal of Pharmaceutical Sciences

Volumn 100, Issue 10, 2011, Pages 4171-4197

  Maddux, Nathaniel R ^a   Joshi, Sangeeta B ^a   Volkin, David B ^a   Ralston, John P ^a   Middaugh, C Russell ^a  

^a UNIVERSITY OF KANSAS   (United States)

Author keywords

Calorimetry; Circular dichroism; Fluorescence; Formulation; Light scattering; Monoclonal antibodies; Phase diagrams; Plasmid DNA; Protein; Stability; Vaccines

Indexed keywords

BETA1A INTERFERON; CLOSTRIDIUM DIFFICILE TOXIN A; CLOSTRIDIUM DIFFICILE TOXIN B; FIBROBLAST GROWTH FACTOR 1; GELATIN; GLYCOPROTEIN GP 96; GRANULOCYTE COLONY STIMULATING FACTOR; HEAT SHOCK COGNATE PROTEIN 70; HEPATITIS C VACCINE; IMMUNOGLOBULIN G; INFLUENZA VACCINE; MALARIA VACCINE; MEASLES VACCINE; MONOCLONAL ANTIBODY; NEUROTOXIN; PHARMACEUTICAL VEHICLES AND ADDITIVES; PRAMLINTIDE; RESPIRATORY SYNCYTIAL VIRUS VACCINE; TYPHOID VACCINE; UVOMORULIN; VACCINE; VELAFERMIN; PROTEIN;

ABSORPTION SPECTROSCOPY; ADENOVIRUS 5; ANALYTIC METHOD; BACILLUS ANTHRACIS; CIRCULAR DICHROISM; CLOSTRIDIUM BOTULINUM TYPE A; DIFFERENTIAL SCANNING CALORIMETRY; DRUG FORMULATION; DRUG MANUFACTURE; EMPIRICAL PHASE DIAGRAM; ENVIRONMENTAL FACTOR; FLUORESCENCE; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HIGH THROUGHPUT SCREENING; HUMAN; INFRARED SPECTROSCOPY; LIGHT SCATTERING; LIPOSOMAL GENE DELIVERY SYSTEM; MATHEMATICAL MODEL; NONHUMAN; NUCLEAR MAGNETIC RESONANCE; RAMAN SPECTROMETRY; REVIEW; TEMPERATURE DEPENDENCE; TYPE III SECRETION SYSTEM; ULTRAVIOLET ABSORBANCE SPECTROSCOPY; ULTRAVIOLET RADIATION; VIRUS LIKE AGENT; X RAY CRYSTALLOGRAPHY; CHEMICAL MODEL; CHEMISTRY; CONFORMATION; DRUG STABILITY; MEDICINAL CHEMISTRY; PHARMACEUTICS; PLASMID; PROCEDURES; PROTEIN CONFORMATION; PROTEIN STABILITY; STATISTICAL MODEL;

ANTIBODIES, MONOCLONAL; BIOPHARMACEUTICS; CHEMISTRY, PHARMACEUTICAL; DRUG STABILITY; HIGH-THROUGHPUT SCREENING ASSAYS; MODELS, CHEMICAL; MODELS, STATISTICAL; NUCLEIC ACID CONFORMATION; PLASMIDS; PROTEIN CONFORMATION; PROTEIN STABILITY; PROTEINS; TECHNOLOGY, PHARMACEUTICAL; VACCINES;

EID: 80052268214     PISSN: 00223549     EISSN: 15206017     Source Type: Journal    
DOI: 10.1002/jps.22618     Document Type: Article

References (81)

1. Pisal DS, Kosloski MP, Balu-Iyer SV. 2010. Delivery of therapeutic proteins. J Pharm Sci 99:2557-2575.
2. Singh SK. 2011. Impact of product-related factors on immunogenicity of biotherapeutics. J Pharm Sci 100:354-387.
3. Lubiniecki A, Volkin DB, Federici M, Bond MD, Nedved ML, Hendricks L, Mehndiratta P, Bruner M, Burman S, DalMonte P, Kline J, Ni A, Panek ME, Pikounis B, Powers G, Vafa O, Siegel R. Comparability assessments of process and product changes made during development of two different monoclonal antibodies. Biologicals 39:9-22.
4. Ramsey JD, Gill ML, Kamerzell TJ, Price ES, Joshi SB, Bishop SM, Oliver CN, Middaugh CR. 2009. Using empirical phase diagrams to understand the role of intramolecular dynamics in immunoglobulin G stability. J Pharm Sci 98:2432-2447.
5. Kueltzo LA, Ersoy B, Darrington T, Ralston JP, Middaugh CR. 2003. Derivative absorbance spectroscopy and protein phase diagrams as tools for comprehensive protein characterization: A bGCSF case study. J Pharm Sci 92:1805-1820.
6. Mach H, Volkin DB, Burke CJ, Middaugh CR. 1995. Ultraviolet absorption spectroscopy. Methods Mol Biol 40:91-114.
7. Lucas LH, Ersoy BA, Kueltzo LA, Joshi SB, Brandau DT, Thyagarajapuram N, Peak LJ, Middaugh CR. 2006. Probing protein structure and dynamics by second derivative ultraviolet absorption analysis of cation-pi interactions. Protein Sci 15:2228-2243.
8. Esfandiary R, Hunjan JS, Lushington GH, Joshi SB, Middaugh CR. 2009. Temperature dependent 2nd derivative absorbance spectroscopy of aromatic amino acids as a probe of protein dynamics. Protein Sci 18:2603-2614.
9. Mach H, Middaugh CR, Lewis RV. 1992. Detection of proteins and phenol in DNA samples with second-derivative absorption spectroscopy. Anal Biochem 200:20-26.
10. Ausar SF, Joshi SB, Middaugh CR. 2008. Spectroscopic methods for the physical characterization and formulation of nonviral gene delivery systems. Methods Mol Biol 434:55-80.
11. Nakanishi K, Berova N, Woody RW. 1994. Circular dichroism-Principles and applications. New York: VCH Publishers Inc.
12. Sreerama N, Manning MC, Powers ME, Zhang JX, Goldenberg DP. 1999. Tyrosine, phenylalanine, and disulfide contributions to the circular dichroism of proteins: Circular dichroism spectra of wild-type and mutant bovine pancreatic trypsin inhibitor. Biochemistry 38:10814-10822.
13. Venyaminov SY, Yang JT. 1996. Determination of protein secondary structure. In Circular dichroism and the conformational analysis of biomolecules; Fasman GD, Ed. New York: Plenum Press, pp 69-107.
14. Jiskoot W, Visser AJWG, Herron JN, Sutter M. 2005. Fluorescence spectroscopy. In Methods for structural analysis of protein pharmaceuticals; Jiskoot W, Crommelin D, Eds. Arlington, Virginia: AAPS Press, pp 27-82.
15. Cardamone M, Puri NK. 1992. Spectrofluorimetric assessment of the surface hydrophobicity of proteins. Biochem J 282:589-593.
16. Stryer L. 1965. The interaction of a naphthalene dye with apomyoglobin and apohemoglobin: A fluorescent probe of non-polar binding sites. J Mol Biol 13:482-495.
17. Guntern R, Bouras C, Hof PR, Vallet PG. 1992. An improved thioflavin S method for staining neurofibrillary tangles and senile plaques in Alzheimer's disease. Experientia 48:8-10.
18. Klunk WE, Pettegrew JW, Abraham DJ. 1989. Quantitative evaluation of congo red binding to amyloid-like proteins with a beta-pleated sheet conformation. J Histochem Cytochem 37:1273-1281.
19. LeVine H. 1993. Thioflavine T interaction with synthetic Alzheimer's disease beta-amyloid peptides: Detection of amyloid aggregation in solution. Protein Sci 2:404-410.
20. LeVine H. 1999. Quantification of beta-sheet amyloid fibril structures with thioflavin T. Methods Enzymol 309:274-284.
21. Demchenko AP. 1988. Red-edge-excitation fluorescence spectroscopy of single-tryptophan proteins. Eur Biophys J 16:121-129.
22. Demchenko AP. 2002. The red-edge effects: 30 years of exploration. Luminescence 17:19-42.
23. Beechem JM, Brand L. 1985. Time resolved fluorescence decay in proteins. Ann Rev Biochem 54:43-71.
24. Alcala JR, Gratton E, Prendergast FG. 1987. Fluorescence lifetime distributions in proteins. Biophys J 41:597-604.
25. Byler DM, Susi H. 1986. Examination of the secondary structure of proteins by deconvoluted FT-IR spectra. Biopolymers 25:469-487.
26. Surewicz WK, Mantsch HH. 1988. New insight into protein secondary structure from resolution enhanced infrared spectra. Biochim Biophys Acta 952:115-130.
27. Aichun D, Ping H, Winslow SC. 1990. Protein secondary structures in water from second-derivative amide I infrared. Biochemistry 29:3303-3308.
28. Thomas GJ. 1999. Raman spectroscopy of protein and nucleic acid assemblies. Ann Rev Biophys Biomol Struct 28:1-27.
29. Freire E. 1995. Differential scanning calorimetry. Methods Mol Biol 40:191-218.
30. Kamerzell TJ, Middaugh CR. 2008. The complex inter-relationships between protein flexibility and stability. J Pharm Sci 97:3494-3517.
31. Cooper A, Johnson CM, Lakey JH, Nollmann M. 2001. Heat does not come in different colours: Entropy-enthalpy compensation, free energy windows, quantum confinement, pressure perturbation calorimetry, solvation and the multiple causes of heat capacity effects in biomolecular interactions. Biophys Chem 93:215-230.
32. Heerklotz PDH. 2007. Pressure perturbation calorimetry. Methods Mol Biol 400:197-206.
33. Sarvazyan AP. 1991. Ultrasonic velocimetry of biological compounds. Ann Rev Biophys Biophys Chem 20:321-342.
34. Berne J, Pecora R. 2000. Dynamic light scattering with applications to chemistry, biology, and physics. New York: Dover.
35. Wiethoff CM, Middaugh CR. 2001. Light-scattering techniques for characterization of synthetic gene therapy vectors. In Nonviral vectors for gene therapy: Methods and protocolsl; Findeis MA, Ed. Totowa, New Jersey: Humana Press Ind, pp 349-376.
36. Irvine GB. 1997. Size-exclusion high-performance liquid chromatography of peptides: A review. Analytica Chimica Acta 352:387-397.
37. Bond MD, Panek ME, Zang Z, Wang D, Mehndiratta P, Zhao H, Gunto K, Ni A, Nedved ML, Burman S, Volkin DB. 2010. Evaluation of a dual-wavelength size exclusion HPLC method with improved sensitivity to detect protein aggregates and its use to better characterize degradation pathways of an IgG1 monoclonal antibody. J Pharm Sci 99:2582-2597.
38. Kissmann J, Ausar SF, Rudolph A, Braun C, Cape SP, Sievers RE, Federspiel MJ, Joshi SB, Middaugh CR. 2008. Stabilization of measles virus for vaccine formulation. Hum Vaccin 4:350-359.
39. Ausar SF, Rexroad J, Frolov VG, Look JL, Konar N, Middaugh CR. 2005. Analysis of the thermal and pH stability of human respiratory syncytial virus. Mol Pharm 2:491-499.
40. Zeng Y, Fan H, Chiueh G, Pham B, Martin R, Lechuga-Ballesteros D, Truong VL, Joshi SB, Middaugh CR. 2009. Towards development of stable formulations of a live attenuated bacterial vaccine: A preformulation study facilitated by a biophysical approach. Hum Vaccin 5:322-331.
41. Rexroad J, Evans RK, Middaugh CR. 2006. Effect of pH and ionic strength on the physical stability of adenovirus type 5. J Pharm Sci 95:237-247.
42. Peek LJ, Brey RN, Middaugh CR. 2007. A rapid, three-step process for the preformulation of a recombinant ricin toxin A-chain vaccine. J Pharm Sci 96:44-60.
43. Rexroad J, Martin TT, McNeilly D, Godwin S, Middaugh CR. 2006. Thermal stability of adenovirus type 2 as a function of pH. J Pharm Sci 95:1469-1479.
44. He F, Joshi SB, Bosman F, Verhaeghe M, Middaugh CR. 2008. Structural stability of hepatitis C virus envelope glycoprotein E1: Effect of pH and dissociative detergents. J Pharm Sci 98:3340-3357.
45. Salnikova MS, Joshi SB, Rytting JH, Warny M, Middaugh CR. 2008. Physical characterization of Clostridium difficile toxins and toxoids: Effect of the formaldehyde crosslinking on thermal stability. J Pharm Sci 97:3735-3752.
46. Markham A, Birket S, Picking WD, Picking WL, Middaugh CR. 2008. pH sensitivity of type III secretion system tip proteins. Proteins: Struct Funct Bioinform 71:1830-1842.
47. Δ5 to temperature and pH. Proteins 73:632-643.
48. Peek LJ, Brandau DT, Jones LS, Joshi SB, Middaugh CR. 2006. A systematic approach to stabilizing EBA-175 RII-NG for use as a malaria vaccine. Vaccine 24:5839-5851.
49. Kissmann J, Joshi SB, Haynes JR, Dokken L, Richardson C, Middaugh CR. 2010. H1N1 influenza virus-like particles: Physical degradation pathways and identification of stabilizers. J Pharm Sci 100:634-645.
50. Ausar SF, Foubert TR, Hudson MH, Vedvick TS, Middaugh CR. 2006. Conformational stability and disassembly of Norwalk virus-like particles: Effect of pH and temperature. J Biol Chem 281:19478-19488.
51. Ruponen M, Braun CS, Middaugh CR. 2006. Biophysical characterization of polymeric and liposomal gene delivery systems using empirical phase diagrams. J Pharm Sci 95:2101-2114.
52. Fan H, Ralston J, DiBase M, Faulkner E, Middaugh CR. 2005. Solution behavior of IFN-β-1a: An empirical phase diagram based approach. J Pharm Sci 94:1893-1911.
53. Nonoyama A, Laurence JS, Garriques L, Qi H, Le T, Middaugh CR. 2008. A biophysical characterization of the peptide drug pramlintide (AC137) using empirical phase diagrams. J Pharm Sci 97:2552-2567.
54. Harn N, Allan C, Oliver C, Middaugh CR. 2007. Highly concentrated monoclonal antibodies: Direct analysis of structure and stability. J Pharm Sci 96:532-546.
55. Brandau DT, Joshi SB, Smalter AM, Kim S, Steadman B, Middaugh CR. 2007. Stability of the Clostridium botulinum type A neurotoxin complex: An empirical phase diagram based approach. Mol Pharm 4:571-582.
56. Fan H, Kashi RS, Middaugh CR. 2006. Conformational lability of two molecular chaperones Hsc70 and GP96: Effects of pH and temperature. Arch Biochem Biophys 447:34-45.
57. Fan H, Li H, Zhang M, Middaugh CR. 2007. Effects of solutes on empirical phase diagrams of human fibroblast growth factor 1. J Pharm Sci 96:1490-1503.
58. Fan H, Vitharana SN, Chen T, O'Keefe D, Middaugh CR. 2007. Effects of pH and polyanions on the thermal stability of fibroblast growth factor 20. Mol Pharm 4:232-240.
59. Jiang G, Joshi SB, Peek LJ, Brandau DT, Huang J, Ferriter MS, Woodley WD, Ford BM, Mar KD, Mikszta JA, Hwang CR, Ulrich R, Harvey NG, Middaugh CR, Sullivan VJ. 2006. Anthrax vaccine powder formulations for nasal mucosal delivery. J Pharm Sci 95:80-96.
60. Esfandiary R, Kickhoefer VA, Rome LH, Joshi SB, Middaugh CR. 2009. Structural stability of vault particles. J Pharm Sci 98:1376-1386.
61. Thyagarajapuram N, Olsen D, Middaugh CR. 2007. The structure stability and complex behavior of recombinant human gelatins. J Pharm Sci 96:3363-3378.
62. Zheng K, Middaugh CR, Siahaan TJ. 2009. Evaluation of the physical stability of the EC5 domain of E-cadherin: Effects of pH, temperature, ionic strength, and disulfide bonds. J Pharm Sci 98:63-73.
63. Gibson TJ, Mccarty K, Mcfadyen IJ, Cash E, DalMonte P, Hinds KD, Dinerman AA, Alvarez JC, Volkin DB. 2011. Application of a high-throughput screening procedure with PEG-induced precipitation to compare relative protein solubility during formulation development with IgG1 monoclonal antibodies. J Pharm Sci 100:1009-1021.
64. Ding K, Louis JM, Gronenborn AM. 2004. Insights into conformation and dynamics of protein GB1 during folding and unfolding by NMR. J Mol Biol 335:1299-1307.
65. Aubin Y, Gingras G, Sauve S. 2008. Assessment of the three-dimensional structure of recombinant protein therapeutics by NMR fingerprinting: Demonstration on recombinant human granulocyte macrophage-colony stimulation factor. Anal Chem 80:2623-2627.
66. Frauenfelder H, Wolynes PG. 1994. Biomolecules: Where the physics of complexity and simplicity meet. Physics Today 47:58-64.
67. Cooper A. 1976. Thermodynamic fluctuations in protein molecules. Proc Natl Acad Sci U S A 73:2740-2741.
68. Stewart GW. 1993. On the early history of the singular value decomposition. SIAM Rev 35:551-566.
69. Chavez JR, Kwarteng AY. 1989. Extracting spectral contrast in Landsat Thematic Mapper image data using selective principal component analysis. Photogrammetric Eng and Remote Sensing 53:339-348.
70. Werring DJ, Clark CA, Barker GJ, Miller DH, Parker GJM, Brammer MJ, Bullmore ET, Giampietro VP, Thompson AJ. 1998. The structural and functional mechanisms of motor recovery: Complementary use of diffusion tensor and functional magnetic resonance imaging in a traumatic injury of the internal capsule. J Neurol Neurosurg Psychiatry 65:863-869.
71. Twellmann T, Saalbach A, Gerstung O, Leach MO, Nattkemper TW. 2004. Image fusion for dynamic contrast enhanced magnetic resonance imaging. BioMed Eng OnLine 3:35.
72. Mansfield JR, Attas M, Majzels C, Cloutis E, Collins C, Mantsch HH. 2002. Near infrared spectroscopic reflectance imaging: A new tool in art conservation. Vibrational Spectrosc 28:59-66.
73. Steiner JE, Menezes RB, Ricci TV, Oliveira AS. 2009. PCA tomography: How to extract information from data cubes. Mon Not R Astron Soc 295:64-75.
74. Hammes GG. 2002. Multiple conformational changes in enzyme catalysis. Biochemistry 41:8221-8228.
75. Kohen A, Cannio R, Bartolucci S, Klinman JP. 1999. Enzyme dynamics and hydrogen tunnelling in a thermophilic alcohol dehydrogenase. Nature 399:496-499.
76. Kraut J. 1988. How do enzymes work? Science 242:533-540.
77. Wand AJ. 2001. Dynamic activation of protein function: A view emerging from NMR spectroscopy. Nat Struct Biol 8:926-931.
78. Kissmann J, Ausar SF, Foubert TR, Brock J, Switzer MH, Detzi EJ, Vedvick TS, Middaugh CR. 2008. Physical stabilization of norwalk virus-like particles. J Pharm Sci 97:4208-4218.
79. Roldao A, Mellado MC, Castilho LR, Carrondo MJ, Alves PM. 2010. Virus-like particles in vaccine development. Expert Rev Vaccines 9:1149-1176.
80. El-Kamary SS, Pasetti MF, Mendelman PM, Frey SE, Bernstein DI, Treanor JJ, Ferreira J, Chen WH, Sublett R, Richardson C, Bargatze RF, Sztein MB, Tacket CO. 2010. Adjuvanted intranasal Norwalk virus-like particle vaccine elicits antibodies and antibody-secreting cells that express homing receptors for mucosal and peripheral lymphoid tissues. J Infect Dis 202:1623-1625.
81. Mackman TJ, Allen CB. 2010. Investigation of an adaptive sampling method for data interpolation using radial basis functions. Int J Numer Meth Eng 83:915-938.