Genome-edited allogeneic CAR T cells
Waseem Qasim (UK) presented research on genome-edited allogeneic CAR T cells. Several clinical trials have shown promising results for CAR T products manufactured from allogeneic transplant donors. However, manufacturing these cells is challenging, even if the cells originate from HLA-matched donors. “Our ambition is to generate these therapies so that they can be used in an off the shelf manner. For that, we apply the emerging genome editing technologies,” said Qasim.
Qasim took the audience through various genome editing tools, including TALEN and CRISPR. He showed results of two trials with UCART19, which is an allogeneic anti-CD19 CAR T cell product produced using TALEN for genome editing. Preliminary data in adult and pediatric B-cell ALL patients are promising, providing proof of concept that an allogeneic off the shelf CAR T product may be feasible. New CRISPR-based technologies are underway; these are expected to reduce the costs and widen the application.