Potency Assays for Advanced Stem Cell Therapy Medicinal Products
Potency Assays for Advanced Stem Cell Therapy Medicinal Products

Author: Jorge S. Burns

Publisher:

Published: 2023

Total Pages: 0

ISBN-13: 9783031300417

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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 .

Potency Assays for Advanced Stem Cell Therapy Medicinal Products
Potency Assays for Advanced Stem Cell Therapy Medicinal Products

Author: Jorge S. Burns

Publisher: Springer Nature

Published: 2023-05-31

Total Pages: 226

ISBN-13: 3031300408

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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

Human Embryonic Stem Cells
Human Embryonic Stem Cells

Author: Arlene Chiu

Publisher: Springer Science & Business Media

Published: 2003-08

Total Pages: 488

ISBN-13:

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A discussion of all the key issues in the use of human pluripotent stem cells for treating degenerative diseases or for replacing tissues lost from trauma. On the practical side, the topics range from the problems of deriving human embryonic stem cells and driving their differentiation along specific lineages, regulating their development into mature cells, and bringing stem cell therapy to clinical trials. Regulatory issues are addressed in discussions of the ethical debate surrounding the derivation of human embryonic stem cells and the current policies governing their use in the United States and abroad, including the rules and conditions regulating federal funding and questions of intellectual property.

Mesenchymal Stem Cell Therapy
Mesenchymal Stem Cell Therapy

Author: Lucas G. Chase

Publisher: Springer Science & Business Media

Published: 2012-12-12

Total Pages: 458

ISBN-13: 1627032002

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Over the past decade, significant efforts have been made to develop stem cell-based therapies for difficult to treat diseases. Multipotent mesenchymal stromal cells, also referred to as mesenchymal stem cells (MSCs), appear to hold great promise in regards to a regenerative cell-based therapy for the treatment of these diseases. Currently, more than 200 clinical trials are underway worldwide exploring the use of MSCs for the treatment of a wide range of disorders including bone, cartilage and tendon damage, myocardial infarction, graft-versus-host disease, Crohn’s disease, diabetes, multiple sclerosis, critical limb ischemia and many others. MSCs were first identified by Friendenstein and colleagues as an adherent stromal cell population within the bone marrow with the ability to form clonogenic colonies in vitro. In regards to the basic biology associated with MSCs, there has been tremendous progress towards understanding this cell population’s phenotype and function from a range of tissue sources. Despite enormous progress and an overall increased understanding of MSCs at the molecular and cellular level, several critical questions remain to be answered in regards to the use of these cells in therapeutic applications. Clinically, both autologous and allogenic approaches for the transplantation of MSCs are being explored. Several of the processing steps needed for the clinical application of MSCs, including isolation from various tissues, scalable in vitro expansion, cell banking, dose preparation, quality control parameters, delivery methods and numerous others are being extensively studied. Despite a significant number of ongoing clinical trials, none of the current therapeutic approaches have, at this point, become a standard of care treatment. Although exceptionally promising, the clinical translation of MSC-based therapies is still a work in progress. The extensive number of ongoing clinical trials is expected to provide a clearer path forward for the realization and implementation of MSCs in regenerative medicine. Towards this end, reviews of current clinical trial results and discussions of relevant topics association with the clinical application of MSCs are compiled in this book from some of the leading researchers in this exciting and rapidly advancing field. Although not absolutely all-inclusive, we hope the chapters within this book can promote and enable a better understanding of the translation of MSCs from bench-to-bedside and inspire researchers to further explore this promising and quickly evolving field.

Regulatory Aspects of Gene Therapy and Cell Therapy Products
Regulatory Aspects of Gene Therapy and Cell Therapy Products

Author: Maria Cristina Galli

Publisher: Springer

Published: 2015-09-15

Total Pages: 235

ISBN-13: 3319186183

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This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Cell and Gene Therapies
Cell and Gene Therapies

Author: Miguel-Angel Perales

Publisher: Springer

Published: 2018-11-27

Total Pages: 286

ISBN-13: 3319543687

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In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.

Encyclopedia of Tissue Engineering and Regenerative Medicine
Encyclopedia of Tissue Engineering and Regenerative Medicine

Author:

Publisher: Academic Press

Published: 2019-06-03

Total Pages: 1436

ISBN-13: 0128137002

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Encyclopedia of Tissue Engineering and Regenerative Medicine, Three Volume Set provides a comprehensive collection of personal overviews on the latest developments and likely future directions in the field. By providing concise expositions on a broad range of topics, this encyclopedia is an excellent resource. Tissue engineering and regenerative medicine are relatively new fields still in their early stages of development, yet they already show great promise. This encyclopedia brings together foundational content and hot topics in both disciplines into a comprehensive resource, allowing deeper interdisciplinary research and conclusions to be drawn from two increasingly connected areas of biomedicine. Provides a ‘one-stop’ resource for access to information written by world-leading scholars in the fields of tissue engineering and regenerative medicine Contains multimedia features, including hyperlinked references and further readings, cross-references and diagrams/images Represents the most comprehensive and exhaustive product on the market on the topic

Reparative Medicine
Reparative Medicine

Author: Jean D. Sipe

Publisher:

Published: 2002

Total Pages: 416

ISBN-13:

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This volume covers topics that encompass the use of components of the body, such as genes, proteins, and cells, to either foster tissue regeneration and remodeling in vivo for the purpose of repairing, replacing, maintaining, or enhancing organ function,

Advances In Pharmaceutical Cell Therapy: Principles Of Cell-based Biopharmaceuticals
Advances In Pharmaceutical Cell Therapy: Principles Of Cell-based Biopharmaceuticals

Author: Christine Guenther

Publisher: World Scientific

Published: 2015-09-23

Total Pages: 565

ISBN-13: 981461680X

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This textbook is a comprehensive overview of the development of cell-based biopharmaceuticals. Beginning with the underlying biology of stem cell and cell-based products, it traces the long and complex journey from preclinical concept to initiation of a pivotal clinical trial and the potential business model behind it.The book also takes into consideration the different regulatory landscapes and their continuous evolution in Europe, North America and other parts of the world. The authors describe a path to manufacture a clinical grade therapeutic that passes all necessary quality measures as a robust and marketable product including an outlook on next generation products and innovative strategies.This reference book is a must-have guide for any professional already active in biopharmaceuticals and anyone interested in getting involved in a scientific, medical or business capacity.

Hepatocyte Transplantation
Hepatocyte Transplantation

Author: S. Gupta

Publisher: Springer Science & Business Media

Published: 2002-09-30

Total Pages: 386

ISBN-13: 9780792387763

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In recent years there has been an increasing need for transplantation, but the number of donor livers available has increased only slightly, despite intensive public relations activities. New concepts in the field of transplantation, for instance the transplantation of living donor organs or the splitting of organs, are urgently required, to safeguard the treatment of patients with severe liver disease. The development and clinical application of cell therapy for patients with liver disease could soon present a significant enhancement of the therapeutic options. The aim of such cell therapy is to repair or improve the biological function of the chronically and acutely damaged liver. Even though systematic trials are not available, individual case reports and small series already show promising clinical results. Present concepts of cell therapy for liver diseases based on the use of primary hepatocytes have recently been considerably extended through new data on the biology of stem cells. The adult haematopoetic stem cell as a pool for hepatocyte grafts - what would be the perspectives for the clinical application? This book is the proceedings of the Falk Symposium No. 126 on `Hepatocyte Transplantation' (Progress in Gastroenterology and Hepatology Part III) held in Hannover, Germany, October 2-3, 2001, and is a forum for basic research, but also for questions concerning clinical applications in the field of hepatocyte transplantation.