Cell and Gene Therapy Manufacturing

Overview

CMC (Chemistry, Manufacturing, and Controls) development for cell and gene therapies, requiring standardization and reproducibility for patient-specific living cell products across a wide range of batch sizes and unpredictable patient scheduling. Manufacturing CMC has been the biggest bottleneck in cell and gene therapy development: making patient-specific, inherently variable living cell products standardized, consistent, and reproducible at appropriate scales is the central challenge. Once a manufacturing process is established for gene therapy, it becomes very difficult to change: even beneficial improvements require comparability studies, investment, and regulatory delays, creating a tension between scientific advancement and manufacturing stability. (Segment 2 (05:00-10:00))