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Index
Cover image Title page Table of Contents Copyright Contributors Proverb Preface Section I: Healthcare and Biopharma Industry, Products and Processes
Chapter 1: Disease and Healthcare Priorities
Abstract 1.1 Introduction 1.2 Status and Measurement of Global Health 1.3 The World is Changing—Demographic Transition 1.4 The World is Changing—Disease Challenge Transition 1.5 Prevention—Health Risks and Their Mitigation 1.6 Priorities for Medicine Development 1.7 Additional Drivers and Challenges in Healthcare
Chapter 2: Brief Review of the Biopharmaceutical and Vaccine Industry
Abstract 2.1 Industrial Context 2.2 Brief History of the Biopharma Industry 2.3 Business Summary 2.4 Biopharma Business Challenges 2.5 Biopharma Pipeline—The Promise
Chapter 3: Selected Biotherapeutics Overview
Abstract 3.1 Introduction 3.2 Marketed Biopharmaceuticals by Molecule Category 3.3 Brief Business Review of Biopharmaceutical Therapeutics 3.4 Current R&D Pipeline of Biotherapeutics and Vaccines 3.5 Basic Processing Characteristics of Biotherapeutics
Chapter 4: Process Capability Requirements
Abstract 4.1 Introduction 4.2 Regulatory Requirements 4.3 Producing the Required Quality 4.4 Producing the Required Quantity 4.5 Product Yield 4.6 Pre-Clinical, Phase I to III Manufacturing, and Regular Production
Section II: Upstream Processes Principles and Methods
Chapter 5: Upstream Bioprocessing: Basic Concepts
Abstract 5.1 Introduction 5.2 Kinetic Models 5.3 A General Mass Balance 5.4 Cell Growth 5.5 Cell Death 5.6 Metabolic Concepts 5.7 Glucose, Glutamine, Lactate and Ammonia 5.8 Oxygen 5.9 Carbon Dioxide 5.10 Product Formation 5.11 Appendix
Chapter 6: Host Cells
Abstract 6.1 Cells in Biopharmaceutical Production 6.2 Animal Cell Culture 6.3 Microbial Systems 6.4 Other Production Systems 6.5 Outlook and Perspective Appendix I The Hybridoma Technology and Humanization of Antibodies
Chapter 7: Cell Line Development
Abstract 7.1 Introduction 7.2 Generation of Stable CHO Cell Lines 7.3 Host Cell Engineering 7.4 Conclusions and Future Perspectives Appendix I Biosafety Analysis for Cell Banks Appendix II Transient Transfection
Chapter 8: Cell Culture Media in Bioprocessing
Abstract 8.1 Introduction 8.2 Cell Culture Media 8.3 Cell Culture Media Components and Supplements 8.4 Serum-Free Media Selection, Development and Optimization 8.5 Manufacturing and Supply of Cell Culture Media 8.6 Quality Systems for Cell Culture Media 8.7 Outsourcing Versus In-House Development and Manufacturing of Cell Culture Media 8.8 Summary
Section III: Recovery Processes, Principles, and Methods
Chapter 9: Industry Review of Cell Separation and Product Harvesting Methods
Abstract 9.1 Introduction 9.2 E. coli System 9.3 Future Developments
Chapter 10: Overview of Alternative Separation Methods in Relation to Process Challenges
Abstract 10.1 How To Become Knowledgeable of Alternative Separations in One Hour 10.2 Overview of Alternative Bioprocessing Operations 10.3 General Comparison of Alternative Separation Methods 10.4 Alternative Separation Operations 10.5 Acknowledgements, Notices, and Disclaimers
Chapter 11: Alternative Separation Methods: Flocculation and Precipitation
Abstract Acknowledgments, Notices, and Disclaimers 11.1 Introduction 11.2 Clarification and Primary Recovery Challenges 11.3 Cell Debris Reduction and Clarification 11.4 Precipitation and Flocculation of Target 11.5 Examples of Contaminant Precipitation 11.6 Sequential and Continuous Precipitation 11.7 Process Economics Notes 11.8 Conclusions
Chapter 12: Alternative Separation Methods: Crystallization and Aqueous Polymer Two-Phase Extraction
Abstract Acknowledgements, Notices, and Disclaimers 12.1 Introduction 12.2 Crystallization 12.3 Aqueous Polymer Two-Phase Extraction 12.4 Crystallization and ATPE—Summing Up
Chapter 13: Expanded Bed Adsorption
Abstract 13.1 Introduction and Background 13.2 Principles of Operation 13.3 Adsorbents for EBA 13.4 Columns for EBA 13.5 Examples 13.6 Cleaning and Sanitization 13.7 Capabilities and Challenges
Chapter 14: Filtration Principles
Abstract 14.1 Introduction 14.2 Types of Filters and MaterialS of Construction 14.3 Nominally-Rated Filters 14.4 Sterilizing-Grade Filters 14.5 Microfiltration Versus Ultrafiltration 14.6 Retention 14.7 Fouling 14.8 Modeling of Filtration Operations 14.9 Summary
Chapter 15: Filtration Methods for Use in Recovery Processes
Abstract 15.1 Introduction 15.2 Normal-Flow Filtration 15.3 Cross-Flow Filtration 15.4 Summary
Section IV: Purification Processes, Principles and Methods
Chapter 16: Introduction to Preparative Protein Chromatography
Abstract 16.1 Introduction 16.2 Chromatographic Principles 16.3 Chromatography Stationary Phase: Properties, Classification, and Basic Concepts 16.4 Mass Transfer Effects in Protein Chromatography 16.5 Modes of Chromatography Operations 16.6 Performance Descriptors for Preparative Protein Chromatography 16.7 Scale-up of Preparative Chromatography 16.8 Economy of Preparative Chromatography 16.9 Purification Strategies 16.10 Introduction to Process Development and Optimization 16.11 Multicolumn Operations 16.12 Conclusions
Chapter 17: Affinity Chromatography
Abstract 17.1 Application Areas 17.2 Examples 17.3 Standard Methods 17.4 Buffers 17.5 CIP 17.6 PD Workflow 17.7 Scale-Up 17.8 Critical Process Parameters 17.9 Economy 17.10 Future Notes
Chapter 18: Ion Exchange Chromatography
Abstract 18.1 Introduction 18.2 Application Areas 18.3 Examples 18.4 IEC Principle and Standard Methods 18.5 Buffers 18.6 Cleaning in Place and Sanitization 18.7 Process Development Workflow 18.8 Critical and Key Process Parameters 18.9 Methodology 18.10 Optimization 18.11 Productivity and Economy 18.12 Future
Chapter 19: Hydrophobic Interaction Chromatography
Abstract 19.1 Application Areas 19.2 Examples 19.3 Standard Methods 19.4 CIP 19.5 PD Workflow 19.6 QbD Perspective 19.7 Methodology 19.8 Economy 19.9 Productivity
Chapter 20: Multimodal Chromatography
Abstract 20.1 Application Areas 20.2 Examples 20.3 Standard Methods 20.4 Buffers 20.5 CIP/SIP 20.6 PD Workflow and Optimization 20.7 Critical Process Parameters 20.8 Economy and Productivity 20.9 Future
Chapter 21: Size Exclusion Chromatography (SEC)
Abstract 21.1 Introduction 21.2 Basic Theory 21.3 Application Areas 21.4 Examples 21.5 Standard Methods 21.6 Buffers 21.7 CIP/SIP 21.8 Process Development (PD) Workflow 21.9 Critical and Key Process Parameters (CPP and KPP) 21.10 Methodology 21.11 Economy/Optimization 21.12 Productivity 21.13 Future
Chapter 22: Reversed Phase Chromatography
Abstract 22.1 Application Areas (Goals and Objectives) 22.2 Example: Insulin Purification 22.3 Standard Methods 22.4 CIP/SIP 22.5 Process Development Workflow 22.6 Critical and Key Process Parameters 22.7 Methodology 22.8 Economy 22.9 Productivity
Chapter 23: Filtration Methods for Use in Purification Processes (Concentration and Buffer Exchange)
Abstract 23.1 Application Areas 23.2 Process Development 23.3 Examples 23.4 Advanced Methods 23.5 Critical Process Parameters (CPP) 23.6 Key Process Parameters 23.7 Economy/Optimization 23.8 Future
Section V: Bioprocessing Equipment
Chapter 24: Upstream Processing Equipment
Abstract 24.1 Introduction 24.2 Common Design Aspects in Bioprocessing Equipment 24.3 The Bioreactor 24.4 Available Bioreactor Technology 24.5 Vessels for Adherent Processes 24.6 High-Throughput Process Development Bioreactors 24.7 Modeling and Simulation
Chapter 25: Downstream Processing Equipment
Abstract 25.1 Introduction 25.2 Critical Aspects of the User Requirement Specification 25.3 Common Components in Equipment for Bioprocessing 25.4 System Flow Path 25.5 Pumps 25.6 Valves 25.7 In-Line Filtration (Sterile Filtration and Particle Filtration) 25.8 Engineering Documents
Chapter 26: Chromatography Columns
Abstract 26.1 Introduction 26.2 Column Design 26.3 Column Packing 26.4 Assessing Column Performance: Efficiency Testing 26.5 Conclusions and Outlook
Chapter 27: Simplification of Buffer Formulation and Improvement of Buffer Control with In-Line Conditioning (IC)
Abstract Acknowledgments 27.1 Introduction 27.2 Buffers for Downstream Processing 27.3 In-Line Dilution (ILD)—Addressing the Footprint Issue 27.4 In-Line Conditioning—Controlled Production of Any Buffer 27.5 Testing and Verifying the IC Buffer Preparation Capabilities 27.6 Straight-Through Processing (STP), an Extension of IC Use
Chapter 28: Continuous Capture of mAbs—Points to Consider and Case Studies
Abstract 28.1 Introduction 28.2 The Rationale for Continuous Processing in Biopharma 28.3 Technical Options for Continuous Purification 28.4 Systems for Continuous Purification 28.5 Process Development Guidance 28.6 Case Studies—Capture and Polishing 28.7 Selected Economic Considerations 28.8 Acknowledgments
Chapter 29: Single Use Technology and Equipment
Abstract 29.1 Introduction 29.2 Overview of Single Use Technologies 29.3 Single Use Material of Construction, Componentry, Assembly, Sterilization, Integrity and Use 29.4 Description of SU Unit Operations and General User Requirement Specifications for a Typical Monoclonal Antibody Suspension Cell Process 29.5 Gaps and Disadvantages of SUT 29.6 Conclusions and the Future of Single Use Technologies
Chapter 30: Process Control and Automation Solutions
Abstract 30.1 Introduction 30.2 Instruments and Input & Outputs 30.3 Automation Hardware 30.4 Programmable Logic Controller & Supervisory Control and Data Access 30.5 Distributed Control System 30.6 The ISA-88 Standard for Batch Control 30.7 Exception Handling 30.8 Computer System Validation 30.9 System Backup, Archival and Disaster Recovery 30.10 Data Historian 30.11 Other Automation Standards 30.12 Further Reading
Section VI: Industrial Process Design
Chapter 31: The Upstream Process: Principal Modes of Operation
Abstract 31.1 Introduction 31.2 Process Development 31.3 Principal Modes of Operation 31.4 The Seed Train 31.5 Process Intensification 31.6 Discussion and Outlook
Chapter 32: Downstream Process Design, Scale-Up Principles, and Process Modeling
Abstract 32.1 Introduction 32.2 Process Design Landscape 32.3 The Core Elements of Process Design 32.4 A Process Design Framework 32.5 Downstream Process Design Methodologies and Tools 32.6 Combining Steps for an Efficient Process 32.7 Scale-up as Part of Process Design 32.8 Downstream 32.9 Process Modeling and Optimization 32.10 Summary
Chapter 33: Cleaning-in-Place and Sanitization
Abstract 33.1 Introduction 33.2 Basic Considerations 33.3 Cleaning and Sanitization of Hardware 33.4 Cleaning and Sanitization of Chromatography Resins 33.5 Cleaning and Sanitization Validation 33.6 Preventive Steps and Bioburden Management 33.7 Economic and Environmental Aspects
Chapter 34: The Search for Process Intensification and Simplification: Alternative Approaches versus Current Platform Processes for Monoclonal Antibodies
Abstract Acknowledgments 34.1 Overview 34.2 Discussion/Conclusions
Chapter 35: Single-Use Technology Implementation For Biologics and Vaccines Production
Abstract Acknowledgments 35.1 Summary 35.2 Benefits of Implementing Single-Use Technology 35.3 Designing and Implementing a Single-Use Technology Process 35.4 Case Studies of Next Generation Processes Enabled by SUT 35.5 Future State Summary
Chapter 36: Points to Consider for Design and Control of Continuous Bioprocessing
Abstract 36.1 Introduction 36.2 Development and Implementation 36.3 Design of Unit Operations 36.4 Integration of Unit Operations 36.5 Summary
Chapter 37: Perfusion N-1 Culture—Opportunities for Process Intensification
Abstract 37.1 Introduction 37.2 N-1 Perfusion Seed Culture 37.3 Available Technology 37.4 Bioreactor Types 37.5 Perfusion Filtration Systems 37.6 Process Development 37.7 Use of Process Analytical Technology (PAT) 37.8 Conclusions 37.9 Future Prospects
Chapter 38: Process Development and Intensification for a Recombinant Protein Expressed in E.coli
Abstract Acknowledgments 38.1 Introduction 38.2 Microbial (E. coli) Expression System and Culture Process Overview 38.3 Cell Line Development 38.4 Culture Process Development and Optimization 38.5 Microbial (E. coli) Downstream Process Overview 38.6 The Baseline Downstream Process and Process Intensification Goals 38.7 Downstream Process Intensification and Improvement 38.8 Summary 38.9 Materials and Methods
Chapter 39: Next-Generation Process Design for Monoclonal Antibody Purification
Abstract Acknowledgments 39.1 Introduction 39.2 Current Practices and Emerging Process Alternatives for Downstream Unit Operations 39.3 Enabling Technologies for Next-Generation Manufacturing Facilities 39.4 Concluding Remarks
Chapter 40: Process Development and Manufacturing of Antibody-Drug Conjugates
Abstract 40.1 Introduction 40.2 Process Development and Manufacturing Considerations 40.3 ADC Conjugation Equipment 40.4 Conclusions
Chapter 41: Process Design for Bispecific Antibodies
Abstract 41.1 Introduction 41.2 Process Designs for Bispecific Antibodies 41.3 Conclusion
Chapter 42: Current Manufacturing of Human Plasma Immunoglobulin G
Abstract 42.1 Introduction 42.2 Plasma Fractionation Technologies 42.3 Processing Technologies to Assure Viral Safety 42.4 Low level Plasma Protein Impurities in Immunoglobulin Products 42.5 Formulation 42.6 Conclusions
Chapter 43: Modern Production Strategies in the Vaccine Industry
Abstract 43.1 Introduction 43.2 Outline 43.3 Whole-Organism Vaccines 43.4 Bacterial Vaccines 43.5 Parasite Vaccines 43.6 Subunit Vaccines 43.7 Polysaccharide and Protein-Polysaccharide Conjugate Vaccines 43.8 Nucleic Acid Vaccines 43.9 Influenza Vaccines 43.10 Vaccine Economics 43.11 Closing Remarks
Chapter 44: Bioprocesses for Cell Therapies
Abstract 44.1 Introduction 44.2 Cell Therapy Bioprocess Economics 44.3 Bioprocess Flowsheet Variations Across Cell Therapies 44.4 Culture Strategies for Cell Therapies 44.5 Differentiation of hPSCs 44.6 Genetic Engineering of Cells for Cell Therapies 44.7 Downstream Processing of Cell Therapies 44.8 Integrated and Continuous Bioprocess Strategies for Cell Therapies 44.9 Manufacturing and Distribution Models for Cell Therapies 44.10 Concluding Remarks
Section VII: Facility Design and Operation
Chapter 45: Facility Design and Process Utilities
Abstract 45.1 Introduction 45.2 The Manufacturing Landscape 45.3 Capacity Planning 45.4 Facility Functional Needs 45.5 cGMP Facility Design 45.6 Multiproduct Manufacturing 45.7 Process Support Functional Considerations 45.8 Planning for the Facility Support Systems on Site 45.9 Facility Construction 45.10 Summary: Considerations When Planning a New Facility Appendix A
Chapter 46: Points to Consider in Manufacturing Operations
Abstract 46.1 Introduction 46.2 Non-GMP Manufacturing 46.3 GMP Manufacturing 46.4 Economics 46.5 Outsourcing
Section VIII: Analytics, Regulatory, Quality, and Safety Aspects
Chapter 47: Analytical Methods
Abstract 47.1 CQA 47.2 Analytical Control Strategy 47.3 Analytical Method Development and Qualification 47.4 Analytical Methods 47.5 Analytical Support of Process Development 47.6 Cost of Operating a Modern, High-Throughput Analytical Laboratory
Chapter 48: Implementation of QbD for Manufacturing of Biologics—Has It Met the Expectations?
Abstract Acknowledgments 48.1 Introduction 48.2 What is QbD? 48.3 Framework for Assessing the Benefits of QbD Application in Manufacturing of Biologics 48.4 Impact of QbD on Biopharmaceutical Life Cycle 48.5 Summary
Chapter 49: Pathogen Safety
Abstract 49.1 Introduction 49.2 Raw Material 49.3 Starting Material 49.4 Capacity of the Manufacturing Process to Inactivate and Remove Pathogens 49.5 Transmissible Spongiform Encephalopathy 49.6 Cleaning and Sanitization of Equipment and Material 49.7 Assessment of Risk of Virus Transmission 49.8 Conclusion Glossary
Chapter 50: Chemistry, Manufacture and Control
Abstract 50.1 Introduction 50.2 Part A: Planning 50.3 Part B: Tech Transfer 50.4 Part C: Execution 50.5 Part D: Common Technical Document Glossary
Chapter 51: Post-Licensure Purification Process Improvements for Therapeutic Antibodies: Current and Future States
Abstract Acknowledgments 51.1 Introduction 51.2 History and Current Status of Therapeutic mAb Production and Purification 51.3 Strategies of Post-Licensure Changes 51.4 Case Studies 51.5 Future Embodiments and Options 51.6 Conclusions
Chapter 52: Navigating the Regulatory Maze Upon Process Changes
Abstract 52.1 Introduction: What is Fragmentation? 52.2 Why Changes Happen: The Business Case for Changes to Approved Processes 52.3 Lack of Common Practice: Impact of Divergent Regulations 52.4 A Call to Action: Against Future Fragmentation
Chapter 53: Security of Bioprocess Consumables Supply
Abstract 53.1 Introduction 53.2 Material and Product Selection 53.3 Supplier Selection 53.4 Risk Management 53.5 Communication 53.6 Conclusion
Section IX: Financial Management and Process Economics
Chapter 54: Basics of Financial Management
Abstract 54.1 Finance Management Principles & Terminology 54.2 Financial Goals and Objectives of Business Enterprises 54.3 Explanation of Financial Key Terms 54.4 Measuring Success
Chapter 55: Management of Process Economy—Case Studies
Abstract Acknowledgments 55.1 Introduction 55.2 Billion-Dollar Questions 55.3 The Objective, the Baseline, and the Return of Improvements 55.4 Facility and Process Cost Break-Down, Outlining the Options 55.5 Million-Dollar Questions 55.6 Financial Analysis—Consider Bias and Transparency 55.7 Conclusions
Section X: Appendices
Chapter 56: Appendix 1—The Ideal-World Research Portfolio
Abstract
Chapter 57: Appendix 2—History of Biomedical Research
Abstract
Chapter 58: Appendix 3—Marketed Biotherapeutics
Abstract
Index
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