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.


Book Synopsis Regulatory Aspects of Gene Therapy and Cell Therapy Products by : Maria Cristina Galli

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Author: Maria Cristina Galli

Publisher:

Published: 2015

Total Pages:

ISBN-13: 9783319186191

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Medical literature for health care practitioners on the evaluation and treatment of breastfeeding issues has been disjointed, conflicting, and difficult to find. The field of breastfeeding medicine itself is nonexistent--there are no "breastfeeding doctors" who are specifically trained to understand this complex and interactive process. While much of the literature about breastfeeding describes how it "should" work, there is currently nothing available to explain why it often fails and how to treat it. Clinician's Guide to Breastfeeding: Evidence-based Evaluation and Management is written for health care practitioners who work with breastfeeding mothers; physicians, nurses, nurse practitioners, and lactation consultants. It provides clear information and clinically tested strategies to help professionals guide new mothers to breastfeed successfully. The first of its kind to consider the entirety of the breastfeeding experience, Clinician's Guide to Breastfeeding is written by Dr. Linda D. Dahl, a leading expert on the subject. It is a comprehensive review of breastfeeding, covering objective analyses of ideal or "normal" nursing, as well as the evaluation and treatment of abnormal nursing, including case studies to illustrate the treatment decision-making process.


Book Synopsis Regulatory Aspects of Gene Therapy and Cell Therapy Products by : Maria Cristina Galli

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by . This book was released on 2015 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: Medical literature for health care practitioners on the evaluation and treatment of breastfeeding issues has been disjointed, conflicting, and difficult to find. The field of breastfeeding medicine itself is nonexistent--there are no "breastfeeding doctors" who are specifically trained to understand this complex and interactive process. While much of the literature about breastfeeding describes how it "should" work, there is currently nothing available to explain why it often fails and how to treat it. Clinician's Guide to Breastfeeding: Evidence-based Evaluation and Management is written for health care practitioners who work with breastfeeding mothers; physicians, nurses, nurse practitioners, and lactation consultants. It provides clear information and clinically tested strategies to help professionals guide new mothers to breastfeed successfully. The first of its kind to consider the entirety of the breastfeeding experience, Clinician's Guide to Breastfeeding is written by Dr. Linda D. Dahl, a leading expert on the subject. It is a comprehensive review of breastfeeding, covering objective analyses of ideal or "normal" nursing, as well as the evaluation and treatment of abnormal nursing, including case studies to illustrate the treatment decision-making process.

Development of Gene Therapies

Development of Gene Therapies

Author: Avery McIntosh

Publisher: Chapman & Hall/CRC Biostatistics Series

Published: 2024-03-13

Total Pages: 0

ISBN-13: 9781032136554

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Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.


Book Synopsis Development of Gene Therapies by : Avery McIntosh

Download or read book Development of Gene Therapies written by Avery McIntosh and published by Chapman & Hall/CRC Biostatistics Series. This book was released on 2024-03-13 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Author: National Academies of Sciences, Engineering, and Medicine

Publisher: National Academies Press

Published: 2020-08-27

Total Pages: 127

ISBN-13: 0309672988

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Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.


Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine

Download or read book Exploring Novel Clinical Trial Designs for Gene-Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Gene and Cell Therapy

Gene and Cell Therapy

Author: Nancy Smyth Templeton

Publisher: CRC Press

Published: 2008-10-06

Total Pages: 1154

ISBN-13: 084938799X

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Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.


Book Synopsis Gene and Cell Therapy by : Nancy Smyth Templeton

Download or read book Gene and Cell Therapy written by Nancy Smyth Templeton and published by CRC Press. This book was released on 2008-10-06 with total page 1154 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Gene Therapy and Cell Therapy Through the Liver

Gene Therapy and Cell Therapy Through the Liver

Author: Shuji Terai

Publisher: Springer

Published: 2015-10-16

Total Pages: 183

ISBN-13: 4431556664

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This book reports the recent progress in gene and cell therapy through the liver and aims to facilitate a comprehensive understanding of the current aspects and future prospects from basic research to clinical therapies. Edited by pioneering researchers, this volume presents extensive information to principal investigators, researchers, postdocs and clinicians for examining the wide varieties of pathological conditions both inside and outside the liver. Providing not only the basic and clinical aspects of therapy, this volume is special in that it focuses on the administrative and regulatory difficulties of actual clinical application and legal regulations in different parts of the globe. By indicating the advantages and limitations of the most promising gene and cell therapies targeting the liver, this book will inspire readers to develop a feasible treatment in the next generation.


Book Synopsis Gene Therapy and Cell Therapy Through the Liver by : Shuji Terai

Download or read book Gene Therapy and Cell Therapy Through the Liver written by Shuji Terai and published by Springer. This book was released on 2015-10-16 with total page 183 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book reports the recent progress in gene and cell therapy through the liver and aims to facilitate a comprehensive understanding of the current aspects and future prospects from basic research to clinical therapies. Edited by pioneering researchers, this volume presents extensive information to principal investigators, researchers, postdocs and clinicians for examining the wide varieties of pathological conditions both inside and outside the liver. Providing not only the basic and clinical aspects of therapy, this volume is special in that it focuses on the administrative and regulatory difficulties of actual clinical application and legal regulations in different parts of the globe. By indicating the advantages and limitations of the most promising gene and cell therapies targeting the liver, this book will inspire readers to develop a feasible treatment in the next generation.

Handbook of Cell and Gene Therapy

Handbook of Cell and Gene Therapy

Author: Hazel Aranha

Publisher: CRC Press

Published: 2023-03-17

Total Pages: 444

ISBN-13: 1000864251

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This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.


Book Synopsis Handbook of Cell and Gene Therapy by : Hazel Aranha

Download or read book Handbook of Cell and Gene Therapy written by Hazel Aranha and published by CRC Press. This book was released on 2023-03-17 with total page 444 pages. Available in PDF, EPUB and Kindle. Book excerpt: This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.

A Handbook of Gene and Cell Therapy

A Handbook of Gene and Cell Therapy

Author: Clévio Nóbrega

Publisher: Springer Nature

Published: 2020-06-27

Total Pages: 195

ISBN-13: 3030413330

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This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field.


Book Synopsis A Handbook of Gene and Cell Therapy by : Clévio Nóbrega

Download or read book A Handbook of Gene and Cell Therapy written by Clévio Nóbrega and published by Springer Nature. This book was released on 2020-06-27 with total page 195 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field.

Cell and Gene Therapies

Cell and Gene Therapies

Author: Miguel-Angel Perales

Publisher: Springer

Published: 2018-11-27

Total Pages: 288

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.


Book Synopsis Cell and Gene Therapies by : Miguel-Angel Perales

Download or read book Cell and Gene Therapies written by Miguel-Angel Perales and published by Springer. This book was released on 2018-11-27 with total page 288 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Oversight and Review of Clinical Gene Transfer Protocols

Oversight and Review of Clinical Gene Transfer Protocols

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2014-03-27

Total Pages: 78

ISBN-13: 030929665X

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Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.


Book Synopsis Oversight and Review of Clinical Gene Transfer Protocols by : Institute of Medicine

Download or read book Oversight and Review of Clinical Gene Transfer Protocols written by Institute of Medicine and published by National Academies Press. This book was released on 2014-03-27 with total page 78 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.