Cellectis, a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), has announced the National Institute of Health’s Recombinant DNA Advisory Committee (RAC)’s unanimous approval of two Phase 1 study protocols for Cellectis’ UCART123, the Company’s most advanced, wholly owned Talen gene edited product candidate in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).
The RAC hearing was held on December 14, 2016 during a session dedicated to UCART projects and Talen based gene editing. This was the first time that allogeneic CAR T-cell programs gene edited with Talen technology were presented during a RAC hearing.
Cellectis said that it expects to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) by the end of 2016 and, pending FDA clearance, plans to initiate Phase 1 clinical trials in the first half of 2017. These programs will be the first therapeutic applications of a gene edited allogeneic “off-the-shelf” product candidate in the U.S.
UCART123 is a gene edited T-cell product candidate that targets CD123, an antigen that is located on CD123-expressing leukemic cells in AML, as well as in leukemic and other tumoral cells in BPDCN.
The clinical research at Weill Cornell will be led by principal investigator Dr. Gail J. Roboz, Director of the Clinical and Translational Leukemia Programs and Professor of Medicine.
The UCART123 clinical program at MD Anderson will be led by Professor Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine and Professor Naveen Pemmaraju, MD, Assistant Professor