Handbook of Cell and Gene Therapy From ProofofConcept 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.FeaturesProvides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editingDiscusses siRNA, mRNA, and plasmid manufacturingDescribes the importance of supplier-sponsor synergies on the path to commercializationWritten for a diverse audience with a large number of individuals in the core technologies and supportive practicesIt 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.