Lessons learned: How can the regenerative medicine field benefit from the experiences in CAR-T cell commercialization?

Written by Miltenyi Biotec

Leading experts in the CAR-T cell field share their experiences in closing commercialization gaps for cell manufacturing approaches.

CAR-T cell therapy might be one of the most promising cellular therapy approaches for some types of hematological cancer1,2. Moving from clinical development towards commercial-scale production, CAR-T cell manufacturing has had to bridge various commercialization gaps. How can the regenerative medicine field benefit from the lessons learned in the CAR-T cell field this far?

What challenges might be faced during the transition from clinical development to commercial-scale production of cellular therapies? What arrangements can be made to increase the speed to market, maybe even support commercial and clinical success? Leading experts in the CAR-T cell field share some valuable insights.

Planning ahead with the right manufacturing strategy

To speed up the time to market, the manufacturing strategy for commercialization should be considered early on. Using research use-only reagents, like media and cytokines, with similar performance as their clinical-grade equivalents in early in vitro or pre-clinical studies could prevent major changes in the production process in later phases. Moreover, you want to keep in mind the scale-up factor, both regarding the reagent supply chain, as well as the manufacturing process. Automated and functionally closed manufacturing processes might help to overcome the hurdles of scaling up and out manual processes, and make them commercially viable. This will facilitate handling various processes in parallel, minimizing the necessary workforce and clean room space. Additionally, automation might reduce operator and process variation. 

Weighing the risk for change

Even with a well-planned manufacturing strategy, the transition from early stage development to commercial-scale manufacturing will require changes in the production process. In this case, understanding your product is key. You should be able to weigh the risk versus the need for change. Knowing how your product works, you will be able to assess safety, functionality and efficacy issues that might arise. In combination with relevant and robust quality assays, this knowledge will give you suitable arguments to convince the regulators.  

What else…?

Listen to the recorded audio of our expert discussion to get more and deeper insights in existing and future ways of how the commercial production of CAR-T cells can succeed:

Advances in CAR-T cell manufacture — Bridging the commercialization gaps


1: Forsberg MH, Das A, Saha K, Capitini CM. The potential of CAR T therapy for relapsed or refractory pediatric and young adult B-cell ALL . Ther. Clin. Risk Manag. 14: 1573—1584 (2018)

2: Maude SL, Laetsch TW, Buechner J et al. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N. Engl. J. Med. 378(5):439—448 (2018)