WU-CART-007 receives orphan drug designation for T-ALL

On March 15, 2022, the allogeneic CD7-directed chimeric antigen receptor (CAR) T-cell product, WU-CART-007, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in the acute lymphoblastic leukemia setting.¹ A phase I/II trial (NCT04984356) evaluating WU-CART-007 for the treatment of patients with relapsed or refractory T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma is underway and the first patient has been dosed.

WU-CART-007 is manufactured using CRISPR/Cas9 technology, allowing the selective deletion of CD7 and the T-cell receptor alpha constant, thereby reducing CAR T-cell fratricide and the risk of graft-versus-host-disease in recipients. If deemed well-tolerated and efficacious in the phase I/II global clinical trial, WU-CART-007 may offer an off-the-shelf CAR T-cell approach for patients with T-cell malignancies.