Demonstrating Drug Potency Early In Cell Therapy Development: Why It's Challenging And Why It Matters

Cell and gene therapies are emerging as an efficacious method for treating complex chronic diseases by using human DNA. However, since the science behind these advanced therapies is fundamentally different from that of their traditional drug counterparts, the development and regulatory process for reviewing these therapies is still evolving.

Advanced therapies are manufactured in patient-specific doses and product manufacturing and quality are more central to the development process, leading the FDA to focus on chemistry manufacturing and controls (CMC) much earlier in development. Sponsors must commit to perfecting the manufacturing of the final product and demonstrating potency much earlier in the development process. Employing the right set of assays is fundamental to provide ample data to support the proposed potency method and maximize potential for regulatory approval.