Feb 7, 2017
Cellectis, the Paris-based biotech, has received an Investigational New Drug, or IND, approval from the U.S. Food and Drug Administration (FDA) for conducting phase I clinical trials with UCART123, the gene-edited product candidate of the biotech, in patients with acute myeloid leukemia, or AML, and blastic plasmacytoid dendritic cell neoplasm, or BPDCN. This is the first “off the shelf” allogeneic gene-edited CAR T-cell product candidate that the FDA has approved for clinical trials. Cellectis anticipates to initiate phase I trials in the first half of 2017.
Dr. Andre Choulika, CEO of Cellectis, said: “We are very excited to have this indication for acute myeloid leukemia, which has approximately 20,000 people getting this cancer every year in the United States and over 10,000 deaths. It’s a very difficult cancer, and we think this product can bring great hope.”
UCART123 is a gene-edited T-cell investigational drug targeting CD123, an antigen expressed at the surface of tumoral cells in BPDCN and leukemic cells in AML. BPDCN is a very rare and aggressive hematological malignancy that is derived from plasmacytoid dendritic cell precursors. It is a disease of bone marrow and blood cells but also often affects skin and lymph nodes.
AML is characterized by uncontrolled accumulation and proliferation of leukemic blasts in bone marrow, peripheral blood and other tissues. These cells disrupt normal hematopoiesis and swiftly cause bone marrow failure and death.