Quality-By-Design Approach To Manufacturing Cell And Gene Therapies

Implementation of a manufacturing process that assures a predefined quality of product is a critical requirement for the licensing and marketing of every cell and gene therapy (CGT) product.

Generally, extensive development is needed to mature an early-stage process to commercial manufacturing because of the biological complexity of the products. However, inadequate process knowledge and understanding constricts implementation of process changes as the impacts on product safety and efficacy are unknown. This often leads to the adoption of processes that, although compliant with established regulations, are not optimal for assuring broad availability to patients who depend on those therapies.

To improve the manufacturing of CGTs, quality-by-design (QbD) principles, widely recognized as integration of scientific knowledge and risk assessment into process and build products, can be adopted. Given the complexity in manufacturing CGTs, incorporating QbD principles at an appropriate stage of development of the product can be significantly advantageous.