Making a Cell Therapy: Principles and Practice of Manufacturing

The success of CAR-T cell therapies, driven by their unprecedented efficacy and recent regulatory approvals, has kick-started a new wave of interest into cells as therapies. Yet manufacturing is the link between an exciting new development in the laboratory and a commercial therapy available to the patients who need it. This course will introduce learners to this new field through the following topics.

• Cellular therapies are living products that are only effective when delivered alive. This course will introduce the basic cellular biology that controls cellular growth and metabolism.
• The enhanced function of some cell therapies relies on our ability to genetically engineer the cells. This course will teach the current and emerging approaches that are used for genetic modification.
• This course will teach how we isolate cells from patients or donors and then manipulate them, including helping those cells to grow and multiply outside of the body, to produce a living product at a therapeutically relevant dose.
• We also need to have confidence that the product we have made is safe and effective and that the process we have used to make it did not encounter any unanticipated obstacles along the way. This course will teach some of the analytical methods used to do this and why these methods were chosen.
• As these are often individualized therapies, how do you reproducibly manufacture a highly complex living therapy for all of the patients who may need it? This course will teach about the unique characteristics of cell therapies and how those characteristics drive manufacturing, delivery, and supply chain decisions.
• Cell therapies are part of the larger pharmaceutical industry, which operates under regulatory oversight. This course will teach about how drugs are regulated and how those regulations are applied specifically to cellular therapies.

This course is intended to provide an overview of the key concepts needed to manufacture a cell therapy for learners interested in entering this exciting new field.