Chimeric antigen receptor (CAR) T cell products are human gene therapy  products in which the T cell specificity is genetically modified to enable recognition of a desired target antigen for therapeutic purposes.  This guidance is intended to assist sponsors, including industry and academic sponsors, developing CAR T cell products.  In this guidance, we, FDA, provide CAR T cell specific recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and clinical study design.  Recommendations specific to autologous or allogeneic CAR T cell products are noted in this guidance.  This guidance also provides recommendations for analytical comparability studies for CAR T cell products.  While this guidance specifically focuses on CAR T cell products, much of the information and recommendations provided will also be applicable to other genetically modified lymphocyte products, such as CAR Natural Killer (NK) cells or T cell receptor (TCR) modified T cells.  These related product types can be highly specialized, and in many cases, considerations beyond those recommended in this guidance would depend on the specific product and manufacturing process.  To discuss considerations specific to these related products, we recommend sponsors communicate with the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) before submitting an Investigational New Drug Application (IND) (e.g., by requesting a pre-IND meeting (Ref. 1)).

Questions?