Handbook of Cell and Gene Therapy: From Proof-of-Concept through Manufacturing to Commercialization PDF
Preview Handbook of Cell and Gene Therapy: From Proof-of-Concept through Manufacturing to Commercialization
Handbook of Cell and Gene Therapy This handbook provides an in-depth review of information across the develop- mental spectrum of gene and cell therapy products. From introductory informa- tion to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formu- lation 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 • 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, scien- tists, management and other academic and research institutions. • Provides the most up-to-date information on the development of gene ther- apy, 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. Handbook of Cell and Gene Therapy From Proof-of-Concept through Manufacturing to Commercialization Edited by Hazel Aranha GAEA Resources Inc., Northport, USA Humberto Vega-Mercado Bristol Myers Squibb, Cell Therapy Operations, Global Manufacturing Sciences and Technology, Warren, USA First edition published 2023 by CRC Press 6000 Broken Sound Parkway NW, Suite 300, Boca Raton, FL 33487-2742 and by CRC Press 4 Park Square, Milton Park, Abingdon, Oxon, OX14 4RN © 2023 selection and editorial matter, Hazel Aranha and Humberto Vega-Mercado; individual chapters, the contributors CRC Press is an imprint of Taylor & Francis Group, LLC Reasonable efforts have been made to publish reliable data and information, but the author and publisher cannot assume responsibility for the validity of all materials or the consequences of their use. The authors and publishers have attempted to trace the copyright holders of all material reproduced in this publication and apologize to copyright holders if permission to publish in this form has not been obtained. If any copyright material has not been acknowledged please write and let us know so we may rectify in any future reprint. Except as permitted under U.S. Copyright Law, no part of this book may be reprinted, reproduced, transmitted, or utilized in any form by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying, microfilming, and recording, or in any information storage or retrieval system, without written permission from the publishers. For permission to photocopy or use material electronically from this work, access www.copyright.com or contact the Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978-750-8400. For works that are not available on CCC please contact mpkbookspermissions@tandf. co.uk Trademark notice: Product or corporate names may be trademarks or registered trademarks and are used only for identification and explanation without intent to infringe. ISBN: 978-1-032-25797-6 (hbk) ISBN: 978-1-032-25798-3 (pbk) ISBN: 978-1-003-28506-9 (ebk) DOI: 10.1201/9781003285069 Typeset in Times by SPi Technologies India Pvt Ltd (Straive) Dedication We would like to dedicate our handbook to the people who motivate and support us in our continued effort to improve health care and treatments across the world: Amy Davis: She provided us with the initial inspiration to pursue this project. Rest in peace Amy! To my late parents, Gregory and Bibiana Aranha, who were educators and recognized the universality of education. Hazel Aranha Money spent on the brain is never spent in vain. – English proverb My wife Donna and our children and grandchildren who are the center of my universe. Humberto Vega Our contributors from across the industry for providing their knowledge, feed- back and guidance. The team at Taylor & Francis for making this a reality. Contents Keywords .............................................................................................................xi Preface ................................................................................................................xiii Author Biography ...............................................................................................xv Contributors ......................................................................................................xvii Abbreviations .....................................................................................................xix Chapter 1 History of Gene Therapy Products ..................................................1 Bridget Heelan Chapter 2 Gene Editing in Humans: How Bioethics Discussions Can Guide Responsible Research and Product Development ..............13 Hazel Aranha Chapter 3 Applying a Risk-Based Approach in the Development and Manufacture of Advanced Therapy Medicinal Products ...............27 Bridget Heelan Chapter 4 Gene Therapy Products: Basics and Manufacturing Considerations ...............................................................................35 Humberto Vega-Mercado Chapter 5 Cell Therapy Products: Basics and Manufacturing Considerations ...............................................................................49 Humberto Vega-Mercado Chapter 6 Facility and Equipment Considerations ........................................65 Humberto Vega-Mercado Chapter 7 Analytical Methods for In-Process Testing and Product Release .............................................................................81 Neil A. Haig, Jamie E. Valeich, Laura DeMaster, Christopher Do, Razvan Marculescu, Jianbin Wang, Bridget S. Fisher, Xiaoping Wu, Akshata Ijantkar, Cayla N. Rodia, Sean D. Madsen, Mohammed Ibrahim, Mel Davis-Pickett, Jennifer F. Hu, Jennifer J. Robbins, James Collins Powell, Divya Shrivastava, and Satyakam Singh vii viii Contents Chapter 8 Validation, Verification and Qualification Considerations ....135 Humberto Vega-Mercado Chapter 9 Adventitious Agent Contamination Considerations during the Manufacture of Cell and Gene Therapy Products ................................................................................153 Hazel Aranha Chapter 10 Training Approaches to Build Cell and Gene Therapy Workforce Capacity ..............................................................175 Orin Chisholm Chapter 11 How to Distribute Cell and Gene Therapies .........................183 Andrea Zobel Chapter 12 Regulatory Compliance and Approval ..................................197 Siegfried Schmitt Chapter 13a Regulatory Landscape in US, EU and Canada .....................209 Kirsten Messmer Chapter 13b Regulatory Landscape in South America .............................225 Heloisa Mizrahy Chapter 13c Regulatory Landscape in Australia and New Zealand ..........233 Orin Chisholm Chapter 13d1 Regulatory Landscape in Singapore .....................................243 Stefanie Fasshauer Chapter 13d2 Regulatory Landscape in Malaysia .......................................255 Stefanie Fasshauer Chapter 13e Regulatory Landscape in China ............................................269 Kai Zhang Contents ix Chapter 13f Regulatory Landscape in Japan ............................................285 Hazel Aranha Chapter 13g Regulatory Landscape in India .............................................295 Arun Bhatt and Rajesh Jain Chapter 14 Avoiding Pitfalls during Advanced Therapy Development .........................................................................311 Kirsten Messmer Chapter 15 Going Forward – Existing and Evolving Technologies (CRISPR, mRNA, siRNA) ...................................................321 Humberto Vega-Mercado and Hazel Aranha Glossary ...........................................................................................................333 Index .................................................................................................................337
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