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
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.