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Investigational New Drug (IND) clearance has been granted by the FDA to proceed with the clinical development of UCART19, an allogeneic, gene-edited cellular therapy candidate to treat relapsed/refractory acute lymphoblastic leukemia.

FDA; acute lymphobla... - Click to enlarge in new windowFDA; acute lymphoblastic leukemia. FDA; acute lymphoblastic leukemia

UCART19 is initially being developed in acute lymphoblastic leukemia and is currently in phase I. The current approach with UCART19 is based on the preliminary positive results from clinical trials using autologous products based on the CAR technology. UCART19 has the potential to overcome the limitation of the current autologous approach by providing an allogeneic, frozen, "off-the-shelf" T-cell-based medicinal product.


The phase I, open-label, dose-escalation CALM study was designed to evaluate safety, tolerability, and antileukemic activity of UCART19 in patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia. With this IND clearance, the CALM study will be expanded to several centers in the U.S., including MD Anderson Cancer Center in Houston.