Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia

Ottaviano and colleagues set out to create a universal T-cell product using genomic editing that would provide an “off the shelf” alternative to current CAR T-cell therapies. Of the six patients who received the study therapy, four went on to receive allogeneic stem cell transplant after experiencing cell expansion and flow cytometric remission. Primary safety objectives were met, with two patients experiencing grade II cytokine release syndrome, one patient developing skin graft-versus-host disease, and one developing transient grade IV neurotoxicity.