Biotechnology and Biopharmaceuticals: Transforming Proteins and Genes into Drugs

The essential single source on drug discovery and biotechnology products—newly revised and updated Since the first edition of Biotechnology and Biopharmaceuticals was published, biotechnology has continued to drive therapeutic product development. The majority of new medicines coming on the market today are developed based on tools created by biotechnology—and healthcare providers who are concerned with optimum drug therapy need to understand the principles underlying the discovery, development, and application of these biological drugs and therapies of the future. Biotechnology and Biopharmaceuticals: Transforming Proteins and Genes into Drugs, Second Edition addresses the pivotal issues relating to translational science, including preclinical and clinical drug development, regulatory science, pharmaco-economics, and cost-effectiveness considerations. It provides research pharmacologists, biopharmaceutical R&D personnel, toxicologists, and biotechnologists with cutting-edge research on new proteins and genetic medicines, the translational and integrated sciences that continue to fuel the innovations in medicine, as well as the new areas of therapeutic development, including cancer vaccines, stem cell therapeutics, and cell-based therapies. Presented in three parts, and containing several appendices, Biotechnology and Biopharmaceuticals : Integrates information from pharmacology, biotechnology, and the medical sciences Covers the science and art of transforming proteins and genes into drugs Offers new coverage of cell-based therapeutics, including stem cells and immune cell therapy Highlights the unique applications of biologics, proteins, and macromolecules as therapeutics Includes new chapters on the clinical trial and approval process of individualized medicine Discusses the future direction of the field Ideal for research pharmacologists, biopharmaceutical R&D personnel, toxicologists, and biotechnologists, Biotechnology and Biopharmaceuticals will also appeal to clinical pharmacologists, pharmacists, physicians, and health scientists.

Contributors Foreword Preface Preface to the First Edition Acknowledgments Organization of the Book User Agreement PART I TRANSFORMING PROTEINS AND GENES INTO DRUGS— THE SCIENCE AND THE ART 1 Introduction to Biopharmaceuticals Abstract 1.1 Background and Significance 1.2 Translation of Biotechnology for Developing Biopharmaceuticals 1.3 Historical Perspective of Pharmaceutical Biotechnology 1.4 Distinctions Between Chemical Drugs Versus Biopharmaceuticals 1.5 Summary 2 Distinctions of Biologic Versus Small Molecule Platforms in Drug Development Abstract 2.1 Introduction 2.2 Transforming New Molecules into Drugs: The Drug Development Process 2.3 Key Differences Between Biotechnology and Chemical Products 2.4 Current Trends in Drug Development 2.5 Summary 3 Financing Biologic Drug Development Abstract 3.1 Introduction 3.2 The Role of the Orphan Drug Act 3.3 Clinical Leverage Strategy in Accelerating Drug Development 3.4 Therapeutic Target Considerations 3.5 Evolving Trends 3.6 Summary 4 Application of Biotechnology in Drug Discovery and Early Development Abstract 4.1 Introduction 4.2 Data Mining, Molecular Cloning, and Characterization 4.3 Optimization of Cell Expression Systems and Product Yield 4.4 Molecular Optimization 4.5 Proteins and Genes as Targets for Drug Discovery and Development 4.6 Summary 5 Large–Scale Production of Recombinant Proteins Abstract 5.1 Introduction 5.2 Yield Optimization in Genetic Constructs and Host Cells 5.3 LargeScale Cultivation of Host Cells 5.4 Downstream Processing and Purification 5.5 Quality Assurance and Quality Control 5.6 Summary 6 Clinical Pharmacology, Toxicology, and Therapeutic Dosage and Response Abstract 6.1 Introduction 6.2 Clinical Pharmacology and Toxicology 6.3 Dose and Therapeutic Response 6.4 Dosage Form and Route of Administration 6.5 Summary 7 Clinical Evaluation and Regulatory Approval and Enforcement of Biopharmaceuticals Abstract 7.1 Introduction: Biologic Drug Development and Approval 7.2 Licensing of Biologic Products 7.3 Preclinical and Clinical Testing 7.4 FDA Review and Approval Process 7.5 Regulatory Enforcement 7.6 Globalization of Drug Approval (Chien) 7.7 Summary 8 Pharmacoeconomics and Drug Pricing (Garrison) Abstract 8.1 Introduction Pharmacoeconomics 8.2 Cost–Effectiveness: Assessing the Value of Biopharmaceuticals 8.3 The Cost of Developing Biopharmaceuticals 8.4 Pricing Biopharmaceuticals 8.5 Drug Development Incentives 8.6 Economics of Biosimilars 8.7 Economic Impact of Personalized Medicine 8.1 Summary and Future Challenges PART II THERAPEUTIC AND CLINICAL APPLICATIONS OF BIOPHARMACEUTICALS— PROTEINS AND NUCLEIC ACIDS 9 Antibodies and Derivatives Abstract Section 1 9.1 Molecular Characteristics and Therapeutic Applications 9.1.1 Introduction 9.1.2 Historical PerspectiveDiscovery, Classification, Biodistribution, and Physiological Characteristics of Antibody Molecules 9.1.3 Gamma Globulin and Hyperimmune Globulin 9.1.4 Monoclonal Antibodies 9.1.5 Antibody Derivatives 9.1.6 Disposition of Antibodies 9.1.7 Extravascular Tissue Penetration 9.1.8 Mechanisms of Antibody Bioactivities 9.1.9 Antibody Conjugates 9.1.10 Antibodies and Derivatives in Development 9.1.11 Summary Section 2 9.2 Antibodies and Derivatives Monographs List 9.3 Antibodies and Derivatives Monographs 10 Hematopoietic Growth and Coagulation Factors Abstract Section 1 10.1 Molecular Characteristics and Therapeutic Applications 10.1.1 Introduction 10.1.2 Hematopoietic Growth and Coagulation Factors 10.1.3 Therapeutic Use of Hematopoietic Growth Factors (Molineux, Elliott) 10.1.4 Therapeutic Use of Coagulation and Anticoagulation Factors 10.1.5 Summary Section 2 10.2 Hematopoietic Growth and Coagulation Factors List 10.3 Hematopoietic Growth and Coagulation Factors 11 Cytokines and Interferons Abstract Section 1 11.1 Molecular Characteristics and Therapeutic Applications 11.1.1 Introduction 11.1.2 Interleukins as Immunotherapeutics 11.1.3 Interferon Biology and Cancer Therapy (Tiu, Koon, Borden) 11.1.4 Interferons as Antiviral Therapy 11.1.5 Interferons in Multiple Sclerosis 11.1.6 Summary Section 2 11.2 Cytokines and Interferons Monographs List 11.3 Cytokines and Interferons Monographs 12 Hormones Abstract Section 1 12.1 Molecular Characteristics and Therapeutic Applications 12.1.1 Introduction 12.1.2 Peptide Hormones and Clinical Significance 12.1.3 Therapeutic Application of Recombinant and Synthetic Hormones 12.1.4 Summary Section 2 12.2 Hormones Monographs List 12.3 Hormones Monographs 13 Enzymes Abstract Section 1 13.1 Molecular Characteristics and Therapeutic Applications 13.1.1 Introduction 13.1.2 Enzyme Replacement Therapy 13.1.3 Enzymes as Therapeutic Agents 13.1.4 Summary and Future Prospects Section 2 13.2 Enzymes Monographs List 13.3 Enzymes Monographs 14 Vaccines (Hu, Ho) Abstract Section 1 14.1 Molecular Characteristics and Therapeutic Applications 14.1.1 Vaccines and Their Impact on Human Health 14.1.2 How Vaccines Work: Interplay among Immune System, Diseases, and Vaccines 14.1.3 Traditional Vaccine Approaches 14.1.4 Subcellular and Recombinant Subunit Vaccines 14.1.5 Vaccine Adjuvants 14.1.6 Mode and Route of Vaccine Delivery 14.1.7 Future Directions 14.1.8 Summary Section 2 14.2 Vaccines Monographs List 14.3 Vaccines Monographs 15 Other Biopharmaceutical Products Abstract 15.1 Other Biopharmaceutical Products Monographs List 15.2 Other Biopharmaceutical Products Monographs PART III FUTURE DIRECTIONS 16 Advanced Drug Delivery Systems Abstract 16.1 Introduction 16.2 Drug Therapeutic Index and Clinical Impact 16.3 Routes of Therapeutic Protein Administration 16.4 Physiological and Mechanistic Approaches 16.5 Approaches Using Devices 16.6 Molecular Approaches 16.7 Summary 17 Advances in Personalized Medicine: Pharmacogenetics in Drug Therapy Abstract 17.1 Introduction to Interindividual Variation 17.2 Historical Perspective on Pharmacogenetics in Drug Safety and Efficacy 17.3 Pharmacogenetics in Drug Disposition and Pharmacokinetics 17.4 Pharmacogenetics in Drug Effects and Pharmacodynamics 17.5 Individualized Gene–Based Medicine: A Mixed Blessing 17.6 Current and Future Prospects of Pharmacogenetics 17.7 Summary 18 Gene and Cell Therapy Abstract 18.1 Overview 18.2 General Strategies in Gene and Cell Therapy 18.3 Gene and Cell Therapy for Select Medical Conditions 18.4 Gene Therapy Research, Development, and Clinical Use (Sullivan) 18.5 Stem Cells in Regenerative Medicine and Diagnostics (Kelly) 18.6 Summary 19 Integration of Discovery and Development: The Role of Genomics, Proteomics, and Metabolomics Abstract 19.1 Overview 19.2 Integration of Discovery and Development of Therapeutic Candidates 19.3 Genomics: The First Link Between Sequences and Drug Targets 19.4 Proteomics: From Sequences to Function 19.5 Metabolomics: Metabolic Profile Elucidation 19.6 Integrating Genomic, Proteomic, and Metabolomic Tools to Accelerate Drug Development 19.7 Summary 20 Pharmacoeconomics, Outcome, and Health Technology Assessment Research in Drug Development Abstract 20.1 Introduction: Health–Care Decisions and Health Outcomes 20.2 Integration of Pharmacoeconomic Outcome Research in Clinical Drug Development 20.3 Regional Differences in the Type of Evidence and Value Data Essential for Health–Care and Reimbursement Decisions 20.4 Biopharmaceutical Company Strategies 20.5 Summary 21 Future Prospects Abstract 21.1 Progress and Benefits in Transforming Proteins and Genes into Biopharmaceuticals 21.2 Genomic Information Improves Safety and Production Cost of Biopharmaceuticals 21.3 The Business of Biopharmaceuticals and Economic Impacts 21.4 Influence of Biopharmaceuticals on Pharmaceutical Research, Development, and the Drug Industry 21.5 Public–Private Partnership in Financial and Regulatory Support to Improve Translational Success 21.6 Biopharmaceuticals and Public Health Benefits 21.7 Public Participation and Influence on Biopharmaceutical Development 21.8 Outlook Appendix I. Dosage Form, Pharmacokinetics, and Disposition Data Appendix II. Molecular Characteristics and Therapeutic Use Appendix III. Nomenclature of Biotechnology Products Appendix IV. Other Information Index