| Description |
1 online resource (372 p.) |
| Contents |
Cover -- Half Title -- Title Page -- Copyright Page -- Dedication -- Table of Contents -- Keywords -- Preface -- Author Biography -- Contributors -- Abbreviations -- Chapter 1: History of Gene Therapy Products -- Historical Timeline and Key Technological Advances -- Key Historical Trials and Lessons Learned -- Key Successes and Regulatory Approvals -- Definition of Gene Therapy in the EU -- Definition of GTMP in the US -- References -- Chapter 2: Gene Editing in Humans: How Bioethics Discussions Can Guide Responsible Research and Product Development -- Introduction |
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Risk-Benefit Considerations -- Bioethics Considerations in Genome Editing -- Regulatory and Nonregulatory Discussions and Declarations -- Concluding Comments -- References -- Chapter 3: Applying a Risk-Based Approach in the Development and Manufacture of Advanced Therapy Medicinal Products -- Introduction -- References -- Chapter 4: Gene Therapy Products: Basics and Manufacturing Considerations -- Introduction -- Manufacturing Process -- Quality Control -- Final Remarks -- References -- Chapter 5: Cell Therapy Products: Basics and Manufacturing Considerations -- Introduction |
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Manufacturing Process -- Final Remarks -- References -- Chapter 6: Facility and Equipment Considerations -- Introduction -- Facility Design for Cell and Gene Therapy Products -- Equipment Design Considerations -- Contract Manufacturing Organizations -- Supplier-Sponsor Synergies in Development of Cell and Gene Therapies -- Final Remarks -- References -- Chapter 7: Analytical Methods for In-Process Testing and Product Release -- Introduction -- General Properties of a Typical CAR T Cell Product -- Design -- MoA and Mode of Action -- CQAs -- Novel CQA Discovery |
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General Considerations for the Control of Product Quality -- Regulatory Expectations for Analytical Procedures -- Development of Analytical Procedures -- Control Strategy for Analytical Procedures -- Documentation of Analytical Procedures in Regulatory Fillings -- Types of Performance Attributes -- Study Designs for the Evaluation of Performance Attributes -- Robustness -- Specificity -- Attribute Specificity -- Representative Material Specificity -- Stability-Indicating Specificity -- Accuracy -- Precision -- Sensitivity -- Linearity -- Range -- Qualifications -- Phase I -- Phase II/III |
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Analytical Life Cycle Management during Clinical Development -- Validations and Verifications -- Analytical Life Cycle Management during Commercial -- Managing Critical Instruments -- Identifying and Managing Critical Reagents -- Continued Performance Verification of Analytical Procedures -- Sample Retention -- Analytical Procedures -- Transduction and Characterization of Integration -- Control of Vector Dose during the Manufacture of a CAR T Cell Product -- Control of Transduction -- qPCR-based Analytical Procedures -- Alternative Technology Platforms |
| Notes |
Description based upon print version of record |
|
Genome Editing and Characterization of Edited Cells |
| Genre/Form |
Electronic books
|
| Form |
Electronic book
|
| Author |
Vega-Mercado, Humberto
|
| ISBN |
9781000864250 |
|
1000864251 |
|
