HANDBOOK OF PHARMACEUTICAL BIOTECHNOLOGY

Edited by SHAYNE COX GAD, PH.D., D.A.B.T. Gad Consulting Services Cary. North Carolina

BICENTENNIAL

18 O7

WILEY
2 OO 7

WILEY-INTERSCIENCE A John Wiley & Sons, Inc., Publication

CONTENTS

Preface

xix

1.1

From Gene to Product: The Advantage of Integrative Biotechnology Frank-Ranter Schmidt Sequencing the Human Genome: Was It Worth It? Eugene Zabarovskv Overview: Differentiating Issues in the Development of Macromolecules Compared with Small Molecules Giinther Blaich, Bernd Janssen, Gregory Roth, and Jochen Salfeld Integrated Development of Glycobiologics: From Discovery to Applications in the Design of Nanoparticular Drug Delivery Systems Christine Vauthier, Isabelle Bertholon, and Denis Labarre R&D Paradigm Shift and Billion-Dollar Biologies Krishan Maggon From Defining Bioinformatics and Pharmacogenomics to Developing Information-Based Medicine and Pharmacotyping in Health Care loannis S. Vizirianakis Toxicogenomics Mary Jane Cunningham and Mrinal Shah

1 53

1.2

1.3

89

1.4

125

1.5

161

201

3.1

229

xiv

CONTENTS

3.2 3.3

Preclinical Pharmacokinetics Donald E. Mager and Murali Ramanthan Strategies for the Cytosolic Delivery of Macromolecules: An Overview Aravind Asokan, Roland Cheung, and Moo J. Cho Basic Issues in the Manufacture of Macromoleucles Corinne Lengsfeld and Thomas Anchordoquy Process Validation for Biopharmaceuticals Beth Junker Stability Assessment and Formulation Characterization Albert H.L. Chow, Henry H.Y. Tong, and Ying Zheng Protein Posttranslational Modification: A Potential Target in Pharmaceutical Development M.D. Mostaqul Huq and Li-Na Wei PEGylation: Camouflage of Proteins, Cells, and Nanoparticles Against Recognition by the Body's Defense Mechanism Yong Woo Cho, Jae Hyung Park, Ji Sun Park, and Kinam Park Unexpected Benefits of a Formulation: Case Study with Erythropoetin David Keast Capillary Separation Techniques Dong Hee Na and Kang Choon Lee Pharmaceutical Bioassay Jun Li, Ming Zhao, and Shiqi Peng Analytical Considerations for Immunoassays for Macromolecules Marian Kelley and Binodh DeSilva Chromatography-Based Separation of Proteins, Peptides, and Amino Acids Jan Moebius, Remco Swart, and Albert Sickman Bioanalytical Method Validation for Macromolecules Gerhard K. E. Scriba Microarrays in Drug Discovery and Development Krishnanand D. Kumble and Sarita Kumble

253

279 297 319 371

4.1 4.2 4.3 4.4

417

4.5

443

4.6

463 469 511

5.1 5.2 5.3

573

5.4

585 611 633

5.5 5.6

CONTENTS 5.7 Genetic Markers and Genotyping Analyses for Genetic Disease Studies Anne E. Kwitek and Michael Olivier Proteins: Hormones, Enzymes, and Monoclonal\Antibodies Background Wayne Materi, Zoltan Gombos, and David S. Wishart Formulation and Delivery Issues of Therapeutic Proteins Sathy V. Balu-Iyer, Razvan D. Miclea, and Vivek S. Purohit Pharmacokinetics Nobuhito Shibata, Yukato ho, and Kanji Takada Immunogenicity of Therapeutic Proteins Suzanne Hermeling, Daan J.A. Crommelin, Huub Schellekens, and Wim Jiskoot Development and Characterization of High-Affinity Anti-Topotecan IgG and Fab Fragments Jin Chen and Joseph P. Balthasar Recombinant Antibodies for Pathogen Detection and Immunotherapy Nicholas J. Pokorny, Jeanine I. Boulter-Bitzer, J. Chris Hall, Jack T. Trevors, and Hung Lee The Radiopharmaceutical Science of Monoclonal Antibodies and Peptides for Imaging and Targeted in situ Radiotherapy of Malignancies Raymond M. Reilly Gene TherapyBasic Principles and the Road from Bench to Bedside Gabor M. Rubanyi Plasmid DNA and Messenger RNA for Therapy Steve Pascolo Formulations and Delivery Limitations of Nucleic-Acid-Based Therapies Tatiana Segura Pharmacokinetics of Nucleic-Acid-Based Therapeutics John C. Schmitz, Aleksandra Pandyra, James Koropatnick, and Randal. W. Berg

xv

661

6.1

691

6.2

737

6.3

757

6.4

815

6.5

835

6.6

851

6.7

883

7.1

943

7.2

971

7.3

1013

7.4

1061

xvi 7.5

CONTENTS Case StudiesDevelopment of Oligonucleotides Ezharul Hoque Chowdhury and Toshihiro Akaike RNA Interference: The Next Gene-Targeted Medicine Andrew V. Oleinikov and Matthew D. Gray Delivery Systems for Peptides/Oligonucleotides and Lipophilic Nucleoside Analogs R.A. Schwendener and Herbert Schott Growth Factors and Cytokines Mandeep K. Mann and Brian E. Cairns Growth Factors, Cytokines, and Chemokines: Formulation, Delivery, and Pharmacokinetics Heping Cao and Rui Lin Protein Engineering with Noncoded Amino Acids: Applications to Hirudin Vincenzo De Filippis Production and Purification of Adenovirus Vectors for Gene Therapy D.M.F. Prazeres and J.A.L. Santos Assessing Gene Therapy by Molecular Imaging Pascal Delepine and Claude Ferec Overview of Stem and Artificial Cells Alejandro Soto-Gutierrez, Nalu Navarro-Alvarez, Jorge David Rivas-Carrillo, and Naoya Kobayashi Regulation of Small-Molecule Drugs Versus Biologicals Versus Biotech Products Maria de los Angeles Cortes Castillo and Jose Luis Di Fabio Intellectual Property and Biotechnology Tania Bubela and Karen Lynne Durell Comparability Studies for Later-Generation Products Plant-Made Pharmaceuticals Patrick A. Stewart Biosimilars H. Schellekens, W. Jiskoot, and D.J.A. Crommelin 1087

7.6

1109

7.7

1149

8.1

1173

8.2

1197

1225

10.1

1261

10.2

1297

11

1313

12.1

1373

12.2

1391

12.3

1433

12.4

1453

CONTENTS

xvii

13.1

The Promise of Individualized Therapy Michael Oettel Enhanced Proteomic Analysis by HPLC Prefractionation Pierre C. Havugimana, Peter Wong, and Andrew Emili An Overview of Metabonomics Techniques and Applications John C. Lindon Bioterrorism Dany Shoham

1463

13.2

1491

13.3

1503

13.4

1525

Index

1653