Handbook of Cell and Gene Therapy : From Proof-of-Concept through Manufacturing to Commercialization book cover
1st Edition

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

  • Available for pre-order on February 20, 2023. Item will ship after March 13, 2023
ISBN 9781032257976
March 13, 2023 Forthcoming by CRC Press
312 Pages 19 Color & 8 B/W Illustrations

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Book Description

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.


  • Intended as a one-stop resource for availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.
  • 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.
  • Diverse audience with a large number of individuals in the core technologies and supportive practices.
  • Table of Contents

    History of Gene Therapy Products; Overview of technologies applied to gene therapy products; History of Cell Therapy Products; Overview of technologies applied to cell therapy products – what it entails; Gene Therapy Manufacturing- an Overview on Viral Vectors and Plasmid Manufacturing, Does it suffice; Basic considerations – Manufacturing Processes: - Cell therapy products; Analytical Methods; Facility and equipment considerations; Control of adventitious agent contamination during manufacture of cell and gene therapy products; Quality Considerations from Clinical to Commercial Manufacturing; Regulatory Compliance and Approval; CMC Submissions; Validation, Verification and Qualification Considerations; Clinical development of cell and gene therapies –from first-in-human to postmarketing studies; Risk-based approach in the development of cell and gene therapy products; Current global regulatory landscape; Comparison of EU and US Regulatory Requirements; Regulatory Landscape for Gene and Cell Therapies; Canada; China; Regulation of cell product and gene therapy; South Asia Countries; Japan; Regulatory Pathway for Cell, Tissue and Gene Therapy Products in Singapore; Regulatory Pathway for Cell and Gene Therapy Products in Malaysia; The Regulation of Cell and Gene Therapy Products in Australia and New Zealand; Pricing and market access: the market access hurdle; Training Approaches to Build Cell and Gene Therapy Workforce Capacity; Lessons Learned – Successes and Pitfalls; Pitfall Avoidance through Cross-Functional Development Approach

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    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).