White Paper: Gene Therapy CMC Strategies Paving the Way for Commercialization

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Earlier days of gene therapy development, and the sometimes dashed hopes of both developers and regulators, taught us that very early-phase product understanding and characterization is critical. Numerous gene therapy developers have moved products through Phases I, Phase II and even into Phase III only to realize that the product was not viable.

This paper discusses early-phase requirements for gene therapy products and the unique challenges gene therapy products present. At several points, the paper will also discuss longer-range CMC challenges and approaches that will ultimately affect the outcome of the development process and the eventual marketability of the product.