The United States Food and Drug Administration (FDA) approved its first Investigational New Drug (IND) submission for a gene-edited chimeric antigen receptor (CAR) T-cell product for clinical trials. CAR T-cell therapy involves using a virus to edit an immune cell (T-cell) extracted from an individual patient, instructing the cell to attack cancer cells within that patient. This autologous process requires individualized treatments for each new patient. Cellectis’ new product (UCART123), which the FDA granted IND approval, is an allogenic CAR-T immunotherapy, meaning it is non-patient specific or “off the shelf.”
Cellectis plans to initiate Phase 1 trials this year. UCART123 targets CD123, an antigen expressed at the surface of leukemic cells in acute myeloid leukemia (AML) and tumorous cells in blastic plasmacytoid dendritic cell neoplasm (BPCDN). AML is characterized by “uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues” and accounts for an estimated 19,950 new cases each year in the U.S alone.1 BPDCN, categorized by the World Health Organization (WHO) under AML, typically presents with features of both lymphoma and leukemia. It impacts bone marrow and blood cells but the most frequently involved site of disease is the skin.2
Pearl Pathways can help your company navigate the complex regulatory landscape towards a pre-IND meeting, new IND filing, 510k submission, and more. Please contact us today to schedule a conversation with our team of industry experts.