Handbook of Cell and Gene Therapy From Proof-of-Concept through Manufacturing to Commercialization

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.

History of Gene Therapy Products Bridget Heelan Ethical Considerations on Cell and Gene Therapies Hazel Aranha Risk-based approach in the development of cell and gene therapy products Bridget Heelan Gene Therapy Products: Basics and manufacturing considerations Humberto Vega Cell therapy products: Basics and manufacturing considerations Humberto Vega Facility and Equipment Considerations Humberto Vega Analytical Methods for in-process testing and product reléase Neil A. Haig Validation, Verification and Qualification Considerations Humberto Vega Control of adventitious agent contamination during manufacture of cell and gene therapy products Hazel Aranha Training Approaches to Build Cell and Gene Therapy Workforce Capacity Orin Chisholm How to distribute Cell and Gene Therapies Andrea Zobel Regulatory Compliance and Approval Siegfried Schmitt (a) Regulatory Landscape in US, EU and Canada Kirsten Messmer (b) Regulatory Landscape in South America Heloisa Mizrahy (c) Regulatory Landscape in Australia and New Zealand Orin Chisholm 13 (d1) Regulatory Landscape in Singapure Stefanie Fasshauer 13 (d2) Regulatory Landscape in Malasya Stefanie Fasshauer 13 (e) Regulatory Landscape in China Kai Zhang 13 (f) Regulatory Landscape in Japan Hazel Aranha 13 (g) Regulatory Landscape in India Arun Bhatt 14. Lesson Learned - Success and Pitfalls (Cross functional Development & The Glycerba Path) Kirsten Messmer 15. Going Forward - Existing and Evolving Technologies (CRISPR, mRNA, siRNA) Hazel Aranha, Humberto Vega

Dr. Hazel Aranha Dr Hazel Aranha is a biopharma professional with over 35 years’ experience in industry, academia and consulting. She is a subject matter expert in the area of adventitious agent contamination in biopharmaceuticals and cell and gene therapy products. Her company, Gaea Resources Inc, has provided consulting and auditing services in the US, Europe, and Asia. Hazel brings deep domain expertise to assist clients in achieving strategic and operational objectives. Her projects have included opportunity mapping and competitive market analysis, due diligence for potential acquisitions, and gap analysis to identify key risks and propose mitigation strategies. She has conducted customized training to address unmet needs in the biopharma sector. Hazel has a Master’s degree in Virology, Ph.D. in Environmental Microbiology and holds Regulatory Affairs Certification (RAC) for both the US and European Union. She has to her credit 2 books, more than 45 publications, and 5 book chapters. She is on the review board of multiple biotechnology journals. Her past assignments have included positions at Sartorius Stedim N.A., Catalent Pharma Solutions, Wyeth Vaccines (Pfizer), and Pall Corporation. She holds professional memberships in the Parenteral Drug Association (PDA) and Regulatory Affairs Professional Society (RAPS). Dr. Humberto Vega-Mercado Affiliation: Bristol Myers Squibb, Cell Therapy Operations, Global Manufacturing Sciences and Technology Dr. Humberto Vega is Sr. Director of BMS., Cell Therapy Operations, Global Manufacturing Sciences and Technology (GMS&T), Head of External Manufacturing Drug Product, Critical Materials, Packaging Technology, Labeling, Cell Therapy Capability Center, Apheresis, and Non-Cell Products Technical Groups based in Summit NJ. He has been in the pharmaceutical and food industries for over 34 years in multiple roles including Research Assistant-Food Processing, Process and Manufacturing Head of sterile and non-sterile pharmaceutical operations, Sr. Scientist and Associate Director of Validation, Technology Head – Vaccine Technology & Engineering, Associate Director of MS&T, and Sr. Director of MS&T. He holds BS and MS degrees in chemical engineering from the University of Puerto Rico and a PhD in engineering science from Washington State University. He holds professional memberships in the Parenteral Drug Association (PDA), Institute of Food Technologists (IFT) and International Society for Pharmaceutical Engineering (ISPE).