Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

B cell acute lymphoblastic leukemia received lymphodepleting chemotherapy and anti-CD52 serotherapy, followed by a single-dose infusion of UCART19 cells. Molecular remissions were achieved within 28 days in both infants, and UCART19 cells persisted until conditioning ahead of successful allogeneic stem cell transplantation. This bridge-to-transplantation strategy demonstrates the therapeutic potential of gene-editing technology.

• W
  Wellcome

  Award: 104807/Z/14/Z
• NI
  National Institute on Handicapped Research

  Award: RP-2014-05-007
• SF
  Seventh Framework Programme

  Award: 602239
• BA
  Biotechnology and Biological Sciences Research Council

  Awards: BB/N003853/1, BB/E005896/1