Quality And Manufacturing System Implications For Cell And Gene Therapy

By Matthew Kanter and Lisa Helmond

While there are slight differences between gene and cell therapy, many of the challenges are the same. Cell therapies or autologous products are those where cells are taken from an individual patient, treated, or expanded in some way, and then returned to the patient they came from to treat their disease. Whereas for gene therapies or allogeneic products, the source material is donor cells. The same is true for in vivo gene therapy where the genes are modified by a vector such as Adeno-associated virus (AAV) to treat or cure a disease. As the industry continues to grow and evolve, advanced therapies are now being produced in unique settings, including hospitals, translational facilities, and facilities for small-scale manufacturing.

In this article, we will examine some of the unique challenges faced by companies that are responsible for manufacturing and ensuring quality for gene and cell therapy products along with potential solutions to improve these processes by using digital manufacturing execution systems (MES) and quality management systems (QMS).