Cellectis, a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), 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 leukaemia (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 expects to file an Investigational New Drug (IND) application with the US 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 US.
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.
AML is a devastating clonal haematopoietic stem cell neoplasm that is characterized by uncontrolled proliferation and accumulation of leukaemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal haematopoiesis and rapidly cause bone marrow failure and death. In the US alone, there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year.
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.
BPDCN is a very rare and aggressive haematological malignancy that is derived from plasmacytoid dendritic cell precursors. BPDCN is primarily a disease of bone marrow and blood cells but also often affects skin and lymph nodes.
The UCART123 clinical programme 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.
Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, engineered CAR T-cells. UCARTs are meant to be readily available CAR T-cells for a large patient population. Their production can be industrialized and standardized with defined pharmaceutical release criteria.
The Recombinant DNA Advisory Committee (RAC) is a federal advisory committee that provides recommendations to the NIH Director related to basic and clinical research involving recombinant or synthetic nucleic acid molecules. The NIH, through the RAC, reviews the most innovative clinical study protocols involving a gene therapy product.