1st Edition
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
- Ethical Considerations on Cell and Gene Therapies
- Risk-based approach in the development of cell and gene therapy products
- Gene Therapy Products: Basics and manufacturing considerations
- Cell therapy products: Basics and manufacturing considerations
- Facility and Equipment Considerations
- Analytical Methods for in-process testing and product reléase
- Validation, Verification and Qualification Considerations
- Control of adventitious agent contamination during manufacture of cell and gene therapy products
- Training Approaches to Build Cell and Gene Therapy Workforce Capacity
- How to distribute Cell and Gene Therapies
- Regulatory Compliance and Approval
- (a) Regulatory Landscape in US, EU and Canada
Bridget Heelan
Hazel Aranha
Bridget Heelan
Humberto Vega
Humberto Vega
Humberto Vega
Neil A. Haig
Humberto Vega
Hazel Aranha
Orin Chisholm
Andrea Zobel
Siegfried Schmitt
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
Biography
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).
Just spreading the exciting news: Humberto Vega, one of the pharmaceutical industry's icon's in the cell and gene therapy space, recently released a book that describes a soup-to-nuts approach to commercialization of cell and gene therapies. It is rare to find any publications in this area that are this comprehensive and cover activities spanning from the early development phase all the way to commercialization. The book is written for a diverse audience that ranges from researchers, scientists and management to academic and research institutions--basically anyone involved in the support of the core technologies and supportive practices in this space. It is widely available for purchase on Amazon, Barnes and Noble, Routledge and many other sources. Congratulations to Humberto Vega and HAZEL ARANHA!
- Brian Kirk
The cell and gene therapies industry is growing rapidly. The "Handbook of Cell and Gene Therapy" is fresh off the presses and contains the latest details on the development, production, and delivery of life-saving cell and gene therapy materials. Co-editor Hazel Aranha, PhD, knows her stuff! Because I edit scientific bioprocessing papers from researchers and commercial production people, I need to stay on top of my knowledge base. This handbook will help me significantly. I am happy to own the book -- as of March 26, 2023 -- and have already started reading it.
- Marcie Brown
