| Description |
1 online resource |
| Series |
Advances in experimental medicine and biology ; v. 1420 |
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Advances in experimental medicine and biology ; v. 1420.
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| Contents |
Intro -- Preface -- Contents -- Contributors -- About the Editor -- 1: The Art of Stem Cell-Based Therapy -- 1.1 The Dawn of a New Era -- 1.2 Lessons from a Past Disease -- 1.3 Potency Assays for Modern Disease Intervention -- 1.4 From Viral Vaccination to Safe Therapy with Cells -- 1.5 From Stem Cell Safety and Efficacy to Potency -- 1.6 Complementing Stem Cell-Based Therapy Art with Standard Operating Procedures -- References -- 2: Potency Assay Development: A Keystone for Clinical Use -- 2.1 Potency Assays as Part of Cell-Based ATMP Quality Control Testing |
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2.1.1 What Is a Potency Assay? -- 2.1.2 Regulatory Requirements for a Potency Assay -- 2.2 Approach to Develop a Potency Assay for Cell-Based ATMPs -- 2.2.1 General Considerations -- 2.2.2 Progressive Implementation of a Potency Assay -- 2.3 Clinical Value of Potency Assays -- 2.3.1 Relationship Between Potency and Clinical Efficacy -- 2.3.2 Potency and Defining an Effective Dose for ATMPs -- 2.4 Potency Assay in Product and Process Development -- 2.4.1 Stability Studies -- 2.4.2 Comparability Studies -- 2.4.3 Compatibility Studies |
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2.5 State of the Art of Potency Assays for Cell-Based ATMPs -- 2.5.1 Examples of Potency Assays for Cell-Based ATMPs -- 2.5.2 Challenges to Potency Assay Development for ATMPs -- 2.6 Conclusion -- References -- 3: Potency Assays: The 'Bugaboo' of Stem Cell Therapy -- 3.1 Potency - What a Cell Can Do -- 3.2 Relevance of Potency Assays -- 3.3 Regulatory Requirements -- 3.3.1 EMA Regulatory Requirements -- 3.3.2 FDA Regulatory Requirements -- 3.3.3 Japanese FDA Regulatory Requirements -- 3.4 Development of Potency Assays -- 3.4.1 Use of Surrogate Markers |
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3.4.2 Autologous and Allogeneic Products -- 3.4.3 Standardisation of Assays -- 3.4.4 Further Considerations -- 3.5 The Quality Target Product Profile -- 3.6 Final Remarks -- References -- 4: Identifying Biomarkers for Osteogenic Potency Assay Development -- 4.1 Skeletal Stem Cell for Conservative Bone Healing -- 4.2 The Challenge of hBM-MSC Donor-Specific Heterogeneity -- 4.3 Telomerised MSC -- Scalable Clonal Populations with Consistent Bone-Forming Potential -- 4.4 Comparative Analysis of Gene Expression, microRNA, Morphological Phenotypes and Cell Membrane or Secreted Proteins |
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4.5 Compelling Pathways for Functional Attributes in Osteogenic Potency Assays -- 4.6 Lessons Learned for Bone Repair ATMP Development -- References -- 5: Potency Assay Considerations for Cartilage Repair, Osteoarthritis and Use of Extracellular Vesicles -- 5.1 Articular Cartilage -- 5.2 Cell-Based Treatment of Cartilage Defects -- 5.2.1 Potency Assays Used for Autologous Chondrocyte Implantation -- 5.2.2 Mechanism of Action (MoA) of MSC-Based Cartilage Defect Repair -- 5.2.3 MoA: Differentiation Versus Paracrine Signalling -- 5.2.4 Cell Fate of MSCs Used to Treat Cartilage Defects |
| Summary |
This volume of the Springer book series Advances in Experimental Medicine and Biology covers potency assays, one of the most complex yet fundamental evaluations that critically influence stem cell regenerative medicine. Developing potency assays for cell-based medicinal products comes with numerous challenges due to the highly specialised nature of the application and purpose. This book provides the reader with the knowledge necessary to understand issues governing the successful development of potency assays, highlighting an international outlook of how the various challenges raised are being managed. Stakeholders concerned with potency assay development range from patient and clinician to contract research organisations, small and medium enterprise, regulatory authorities and even politicians. The value of potency assays is poised to increase given the inevitable watershed as early-stage clinical trials addressing safety progress to trials testing efficacy. Contributors from clinical, academic, industrial and regulatory sectors establish a broad point of view for guidance and timely debate. Potency assays require extensive collaboration across disciplines and sectors, as well as compromise and the authors aim to constructively address the many key aspects involved. Potency assays provide a quantitative measure of the biological activity of advanced therapy medicinal products (ATMPs) and thus are required for their market authorization. As the pace of ATMP development accelerates, the need to develop specific, accurate, and robust potency assays for each product is also accelerating. The volume Potency Assays for Stem Cell Advanced Therapy Medicinal Products presents a broad outlook on the development, quality attributes, and implementation of potency assays for ATMPs. The first few chapters introduce a nuanced historical perspective on the science of potency assay development, describe specific quality attributes of an idealized potency assay, indicate pitfalls associated with developing such assays for ATMPs, and review guidance recommended by regulatory authorities on assay suitability for product approval. Subsequent chapters highlight efforts to develop potency assays for specific ATMPs, including skeletal stem cells, mesenchymal stromal cells, extracellular vesicles, CAR T-cells, and discuss emerging technologies/platforms for potency assay design. The volume concludes with a chapter reviewing potency assays used for the release of commercial ATMP products, which amalgamates information contained in previous chapters. Overall, the knowledge contributed from leading authorities in both academia and industry is an ideal resource for technicians, scientists, clinicians, process engineers, and regulators working with ATMPs. Donald G. Phinney, PhD Professor, Department of Molecular Medicine, Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology |
| Analysis |
Biology |
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Science |
| Bibliography |
Includes bibliographical references and index |
| Notes |
Online resource; title from PDF title page (SpringerLink, viewed June 13, 2023) |
| Subject |
Stem cells -- Therapeutic use
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| Form |
Electronic book
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| Author |
Burns, Jorge S
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| ISBN |
9783031300400 |
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3031300408 |
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