GMP Principles: The True Star Of Cell & Gene FDA Interactions

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

Though many topics caught my attention during the FDA’s recent cell and gene therapy town halls, I was struck by the sheer number of questions related to defining, demonstrating, and documenting the quality of our raw materials.

The industry’s focus on raw material quality shouldn’t necessarily come as a surprise, seeing as the FDA itself acknowledged that a common CMC issue causing clinical holds for Phase 1 studies includes “using reagents which are not demonstrated to be of a sufficient safety or quality.” But I daresay the regulators themselves were slightly surprised by just how many questions we posed about our raw material quality, use, and documentation.