An Empirical Phase Diagram-High-Throughput Screening Approach to the Characterization and Formulation of Biopharmaceuticals

Formulation and Process Development Strategies for Manufacturing Biopharmaceuticals

Volumn , Issue , 2010, Pages 173-205

  Joshi, Sangeeta B ^a   Bhambhani, Akhilesh ^a   Zeng, Yuhong ^a   Middaugh, C Russell ^a  

^a UNIVERSITY OF KANSAS   (United States)

Author keywords

Empirical phase diagrams using single technique or multiple independent techniques; EPD approach, for rapid preformulation of vaccines and macromolecule based pharmaceuticals; High throughput screening approach characterization and formulation of biopharmaceuticals

Indexed keywords

EID: 84855878469     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9780470595886.ch8     Document Type: Chapter

References (58)

1. Chang, B. S. and Hershenson, S. (2002), Practical approaches to protein formulation development, in Rational Design of Stable Protein Formulations: Theory and Practice, Carpenter, J. F. and Manning, M. C., eds., Kluwer Academic, New York, pp. 1-25.
2. Venyaminov, S. Y. and Yang, J. T. (1996), Determination of protein secondary structure, in Circular Dichroism and the Conformational Analysis of Biomolecules, Fasman, G. D., ed., Plenum Press, New York, pp. 69-107.
3. Fan, H., Vitharana, S. N., Chen, T., O'Keefe, D., and Middaugh, C. R. (2007), Effects of pH and polyanions on the thermal stability of fibroblast growth factor 20, Mol. Pharm. 4: 232-240.
4. Ausar, S. A., Joshi, S. B., and Middaugh, C. R. (2008) Spectroscopic methods for the physical characterization and formulation of nonviral gene delivery systems, in Methods in Molecular Biology: Gene Therapy Protocols, (Le Doux, J. M. ed.) Humana Press, Totowa, NJ. pp. 55-80.
5. Braun, C. S., Kueltzo, L. A., and Middaugh, C. R. (2001), Ultraviolet absorption and circular dichroism spectroscopy of nonviral gene delivery complexes, in Nonviral Vectors for Gene Therapy: Methods and Protocols, Findeis, M. A., ed., Humana Press, Totowa, NJ, pp. 253-284.
6. Jiskoot, W., Visser, A. J. W. G., Herron, J. N., and Sutter, M. (2005), Fluorescence spectroscopy, in Methods for Structural Analysis of Protein Pharmaceuticals, Jiskoot, W. and Crommelin, D., eds., American Association of Pharmaceutical Scientists (AAPS), pp. 27-82.
7. 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.
8. Cardamone, M. and Puri, N. K. (1992), Spectrofluorimetric assessment of the surface hydrophobicity of proteins, Biochem. J. 282(Pt. 2): 589-593.
9. Ali, V., Prakash, K., Kulkarni, S., Ahmad, A., Madhusudan, K. P., and Bhakuni, V. (1999), 8-anilino-1-naphthalene sulfonic acid (ANS) induces folding of acid unfolded cytochrome c to molten globule state as a result of electrostatic interactions, Biochemistry 38: 13635-13642.
10. Fan, H., Kashi, R. S., and Middaugh, C. R. (2006), Conformational lability of two molecular chaperones Hsc70 and GP96: Effects of pH and temperature, Arch. Biochem. Biophys. 447: 34-45.
11. Matulis, D. and Lovrien, R. (1998), 1-Anilino-8-naphthalene sulfonate anion-protein binding depends primarily on ion pair formation, Biophys. J. 74: 422-429.
12. Klunk, W. E., Pettegrew, J.W., and Abraham, D. J. (1989), Quantitative evaluation of congo red binding to amyloid-like proteins with a beta-pleated sheet conformation, J. Histochem. Cytochem. 37: 1273-1281.
13. Guntern, R., Bouras, C., Hof, P. R., and Vallet, P. G. (1992), An improved thioflavine S method for staining neurofibrillary tangles and senile plaques in Alzheimer's disease, Experientia 48: 8-10.
14. LeVine, H., 3rd. (1993), Thioflavine T interaction with synthetic Alzheimer's disease betaamyloid peptides: Detection of amyloid aggregation in solution, Protein Sci. 2: 404-410.
15. LeVine, H., 3rd. (1999), Quantification of beta-sheet amyloid fibril structures with thioflavin T, Meth. Enzymol. 309: 274-284.
16. Vassar, P. S. and Culling, C. F. (1959), Fluorescent stains, with special reference to amyloid and connective tissues, Arch. Pathol. 68: 487-498.
17. Alonso, L. G., Garcia-Alai, M. M., Smal, C., Centeno, J. M., Iacono, R., Castano, E., Gualfetti, P., and de Prat-Gay, G. (2004), The HPV16 E7 viral oncoprotein self-assembles into defined spherical oligomers, Biochemistry 43: 3310-3317.
18. Hatters, D. M., MacPhee, C. E., Lawrence, L. J., Sawyer, W. H., and Howlett, G. J. (2000), Human apolipoprotein C-II forms twisted amyloid ribbons and closed loops, Biochemistry 39: 8276-8283.
19. Wiethoff, C. M. and Middaugh, C. R. (2001), Light-scattering techniques for characterization of synthetic gene therapy vectors, in Nonviral Vectors for Gene Therapy: Methods and Protocols, Findeis, M. A., ed., Humana Press, Totowa, NJ, pp. 349-376.
20. Freire, E. (1995), Differential scanning calorimetry, Meth. Mol. Biol. 40: 191-218.
21. Kueltzo, L. A., Ersoy, B., Darrington, T., Ralston, J. P., and Middaugh, C. R. (2003), Derivative absorbance spectroscopy and protein phase diagrams as tools for comprehensive protein characterization: A bGCSF case study, J. Pharm. Sci. 92: 1805-1820.
22. Kueltzo, L. A. and Middaugh, C. R. (2003), Structural characterization of bovine granulocyte colony stimulating factor: Effect of termperature and pH, J. Pharm. Sci. 92: 1793-1804.
23. Fan, H., Ralston, J., DiBase, M., Faulkner, E., and Middaugh, C. R. (2005), Solution behavior of IFN-ß-1a: An empirical phase diagram based approach, J. Pharm. Sci. 94: 1893-1911.
24. Jiang, G., Joshi, S. B., Peek, L. J., Brandau, D. T., Huang, C. R., Ferriter, M. S., Woodley, W. D., Ford, B. M., Mar, K. D., Mikszta, J. A., Hwang, C. R., Ulrich, R., Harvey, N. G., Middaugh, C. R., and Sullivan, V. J. (2006), Anthrax vaccine powder formulations for nasal mucosal delivery, J. Pharm. Sci. 95: 80-96.
25. Peek, L. J., Brey, R. N., and Middaugh, C. R. (2007). A rapid, three-step process for the preformulation of a recombinant ricin toxin A-chain vaccine, J. Pharm. Sci. 96: 44-60.
26. Fan, H., Li, H., Zhang, M., and Middaugh, C. R. (2007), Effects of solutes on empirical phase diagrams of human fibroblast growth factor 1, J. Pharm. Sci. 96: 1490-1503.
27. Brandau, D. T., Joshi, S. B., Smalter, A. M., Kim, S., Steadman, B., and Middaugh, C. R. (2007), Stability of the Clostridium botulinum type A neurotoxin complex: An empirical phase diagram based approach, Mol. Pharm. 4: 571-582.
28. Ausar, S. F., Rexroad, J., Frolov, V. G., Look, J. L., Konar, N., and Middaugh, C. R. (2005), Analysis of the thermal and pH stability of human respiratory syncytial virus, Mol. Pharm. 2: 491-499.
29. Ausar, S. F., Foubert, T. R., Hudson, M. H., Vedvick, T. S., and C.R., M. (2006), Conformational stability and disassembly of Norwalk virus like particles: Effect of pH and temperature, J. Biol. Chem. 281: 19478-19488.
30. Harn, N., Allan, C., Oliver, C., and Middaugh, C. R. (2007), Highly concentrated monoclonal antibodies: Direct analysis of structure and stability, J. Pharm. Sci. 96: 532-546.
31. Peek, L. J., Brandau, D. T., Jones, L. S., Joshi, S. B., and Middaugh, C. R. (2006), A systematic approach to stabilizing EBA-175 RII-NG for use as a malaria vaccine, Vaccine 24: 5839-5851.
32. Rexroad, J., Martin, T. T., McNeilly, D., Godwin, S., and Middaugh, C. R. (2006), Thermal stability of adenovirus type 2 as a function of pH, J. Pharm. Sci. 95: 1469-1479.
33. Ruponen, M., Braun, C. S., and C.R., M. (2006), Biophysical characterization of polymeric and liposomal gene delivery systems using empirical phase diagrams, J. Pharm. Sci. 95: 2101-2114.
34. Thyagrajapuram, N., Olsen, D., and C.R.,M. (2007), The structure stability and complex behavior of recombinant human gelatins, J. Pharm. Sci. 96: 3363-3378.
35. Nonoyama, A., Laurence, J. S., Garriques, L., Qi, H., Le, T., and Middaugh, C. R. (2008), A biophysical characterization of the peptide drug pramlintide (AC137) using empirical phase diagrams, J. Pharm. Sci., 97: 2552-2567.
36. Salnikova, M. S., Joshi, S. B., Rytting, J. H., Warny, M., and Middaugh, C. R. (2008), Physical characterization of Clostridium difficile toxins and toxoids: Effect of the formaldehyde crosslinking on thermal stability, J. Pharm. Sci. 97: 3735-3752.
37. Rexroad, J., Evans, R. K., and Middaugh, C. R. (2006), Effect of pH and ionic strength on the physical stability of adenovirus type 5, J. Pharm. Sci. 95: 237-247.
38. Barrett, B. S., Picking, W. L., Picking, W. D. and Middaugh, C. R. (2008), The response of type three secretion system needle proteins MxiHDelta5, BsaLDelta5, and PrgIDelta5 to temperature and pH, Proteins 73(3): 632-643.
39. He, F., Joshi, S. B., Moore, D. S., Shinogle, H. E., Ohtake, S., Lechuga-Ballesteros, D., Martin, R. A., Truong-Le, V. L. and Middaugh, C. R. (2010), Using spectroscopic and microscopic methods to probe the structural stability of human adenovirus type 4, Human Vaccines 6(2): 204-211.
40. Kissmann, J., Ausar, S. F., Rudolph, A., Braun, C., Cape, S. P., Sievers, R. E., Federspiel, M. J., Joshi, S. B., and Middaugh, C. R. (2008), Stabilization of measles virus for vaccine formulation, Human Vaccines 4(5): 350-359.
41. Esfandiary, R., Yee, L., Ohtake, S., Martin, R. A., Truong-Le, V. L., Laurence, J. S., Lechuga-Ballesteros, D., Moore, D. S., Joshi, S. B., and Middaugh, C. R. (2010), Biophysical Characterization of Rotavirus Serotypes G1, G3, and G4, Human Vaccines 6(5): (in press)
42. Esfandiary, R., Kickhoefer, V. A., Rome, L. H., Joshi, S. B., and Middaugh, C. R. (2009), Structural stability of vault particle, J. Pharm. Sci. 98(4): 1376-1386.
43. Zeng, Y., Fan, H., Chiueh, G., Pham, B., Martin, R., Lechuga-Ballesteros, D., Truong, V. L., Joshi, S. B., and Middaugh, C. R. (2009), Towards development of stable formulations of a live attenuated bacterial vaccine: a preformulation study facilitated by a biophysical approach. Human Vaccinces 5(5): 322-331.
44. Shire, S. J., Shahrokh, Z., and Liu, J. (2004), Challenges in the development of high protein concentration formulations, J. Pharm. Sci. 93: 1390-1402.
45. Zimmer, C., Luck, G., and Venner, H. (1968), Studies on the conformation of protonated DNA, Biopolymers 6: 563-564.
46. Kamerzell, T. J. and Middaugh, C. R. (2007), Two-dimensional correlation spectroscopy reveals coupled immunoglobulin regions of differential flexibility that influence stability, Biochemistry 46: 9762-9773.
47. Miller, D. W. and Dill, K. A. (1995), A statistical mechanical model for hydrogen exchange in globular proteins, Protein Sci. 4: 1860-1873.
48. Woodward, C. K. and Hilton, B. D. (1979), Hydrogen exchange kinetics and internal motions in proteins and nucleic acids, Annu. Rev. Biophys. Bioeng. 8: 99-127.
49. Joshi, S. B., Kamerzell, T. J., McNown, C., and Middaugh, C. R. (2008), The interaction of heparin/polyanions with bovine, porcine, and human growth hormone, J. Pharm. Sci. 97: 1368-1385.
50. Eftink, M. and Ghiron, C. (1975), Dynamics of a protein matrix revealed by fluorescence quenching, Proc. Natl. Acad. Sci. USA 72: 3290-3294.
51. Kamerzell, T. J., Unruh, J. R., Johnson, C. K., and Middaugh, C. R. (2006), Conformational flexibility, hydration and state parameter fluctuations of fibroblast growth factor-10:effects of ligand binding, Biochemistry 45: 15288-15300.
52. Ramsey, J. D., Gill, M. L., Kamerzell, T. J., Price, E. S., Joshi, S. B., Bishop, S. M., Oliver, C. N., and Middaugh, C. R. (2009), Using empirical phase diagrams to understand the role of intramolecular dynamics in immunoglobulin G stability, J. Pharm. Sci. 98(7): 2432-2447.
53. Maclean, D. S., Qian, Q., and Middaugh, C. R. (2002), Stabilization of proteins by low molecular weight multi-ions, J. Pharm. Sci. 91: 2220-2229.
54. Yoshioka, S. and Stella, V. J. (2000), Stability of Drugs and Dosage Forms, Kluwer Academic/ Plenum Publishers, New York, pp. 187-199.
55. Ausar, S. F., Espina, M., Brock, J., Thyagarayapuran, N., Repetto, R., Khandke, L., and Middaugh, C. R. (2007), High-throughput screening of stabilizers for respiratory syncytial virus: Identification of stabilizers and their effects on the conformational thermostability of viral particles, Human Vaccines 3: 94-103.
56. Salnikova, M. S., Joshi, S. B., Rytting, J. H., Warny, M., and Middaugh, C. R. (2008), Preformulation studies of Clostridium difficile toxoids A and B, J. Pharm. Sci. 97: 4194-4207.
57. Markham, A., Birket, S., Picking, W. D., Picking, W. L., and Middaugh, C. R. (2008), pH sensitivity of type III secretion system tip proteins, in Proteins: Structure, Function and Bioinformatics, 71: 1830-1842.
58. Kissmann, J., Ausar, S. F., Foubert, T. R., Brock, J., Switzer, M. H., Detzi, E. J., Vedvick, T. S., and Middaugh, C. R. (2008), Physical stabilization of Norwalk virus-like particles, J. Pharm. Sci. 97: 4208-4218.