Cell and gene therapies promise to transform treatment for many lethal and debilitating diseases. It will also change biomanufacturing. This presentation will discuss the evolution of the cell and gene therapy landscape over the coming decade, and the associated opportunities and challenges for manufacturers and their suppliers.

By considering the actual process operations for an autologous cell therapy, the design of a manufacturing facility can help improve the cost of goods (COGS) for the product. This can be achieved mainly by increasing the capacity of the facility, while maintaining a small footprint. This presentation will discuss the possibilities and requirements for the design of a cost-effective facility.

The era of gene editing has unlocked the potential to cure diseases with few treatment options. In particular, gene-edited stem cell therapies have shown great promise in the treatment of beta-thalassemia and sickle cell disease, and could change the treatment landscape in hematological malignancies. This talk will discuss some points to consider when developing a gene-edited stem cell manufacturing process.

Immatics utilizes target discovery platform, XPRESIDENT, which identifies tumor targets and screens cognate TCRs for off-target toxicities. TCRs against these tumor targets are used in various Immatics’ adoptive cellular therapy programs that include endogenous autologous, engineered autologous, and engineered
allogeneic products for various cancer indications. Extensive process development led to the manufacturing approaches for the various product platforms, and exemplary manufacturing and/or clinical data from various trials will be presented for the various platforms.

CAR T cells have been a major focus of investigators, but other targeting strategies have demonstrated similar promise for treating viral infections and tumors. Here, we will discuss the ex vivo expansion of antigen-specific T cells, and how they contrast
with CAR T cells. More specifically, we will evaluate starting materials, manufacturing time, product variability, and process development needs.

Making a decision on where and how to manufacture a therapy is one of the most important decisions a company will make. The decision has impact on both scientific and clinical strategies, as well as fundraising/capital strategies. Criteria for considering how to make the most informed decision will be reviewed with case studies.

Adoptive immunotherapy with ex vivo expanded antigen-specific T cells has shown promise for patients with various cancers and viral infections. However, current manufacturing processes limit the cell yield and potential clinical applications to these investigational therapies. In this presentation, we will review how the Cellular Therapy Laboratory at the Children’s National Hospital addresses the need to scale up cell therapy manufacturing by using higher-capacity cell culture strategies and automated systems.

Analysis of the metabolome can be used for the evaluation of pluripotent and multipotent stem cell cultures, affording early detection of physiological changes indiscernible with traditional culture monitoring techniques. Finally, metabolomics can be used for the evaluation of the quality of stem cell differentiation in 2D and 3D biomaterial cultures, providing a sensitive and robust state-of-the-art technology, guaranteeing high-quality cell therapy manufacturing.