Cell and gene therapy is a relatively new therapeutic area particularly in the regulatory space. However, regulatory agencies are rapidly gaining experience with the approval and marketing of CGT products. While several guidance documents have been published to aid in the development of CGT products, the regulatory aspects of CGT are still evolving to keep pace with scientific and technology advancements.
Inherent properties of CGT products such as short shelf life (autologous), use of novel technologies, complex characterization, and unknown mechanisms of action (MOA) pose challenges in CMC development. In addition, complex manufacturing processes and complicated supply chain/logistics with accelerated product development timelines, all contribute to CMC hurdles that sponsors will likely face. The adage that the process is the product, so true for conventional biologics, is even more critical for CGT development. The importance of CMC in CGT development is evident from the greater amount of CMC information requests (IRs) received by sponsors during regulatory review. News of delayed marketing of some CGT products because of CMC related issues also highlight the importance of getting CMC right early in development.