The FDA has gone on to publish its final guidance on January 30, 2024, that goes on to offer consideration for the development of chimeric antigen receptor- CAR T-cell products. As per the FDA, CAR T-cell products happen to be human gene therapy products wherein the T cell specificity happens to be genetically modified so as to enable recognition of target antigen for therapeutic purposes that’s desired.
The guidance goes on to offer sponsors data so as to help create CAR T-cell products, which include specific recommendations with regards to chemistry, manufacturing, as well as controls- CMC, pharmacology, and toxicology, and also clinical study design. Considerations when it comes to autologous or allogeneic CAR T-cell products happen to be addressed, as well as analytical comparability studies.
The offered recommendations also happen to be applicable to other genetically modified lymphocyte products, such as CAR natural killer cells as well as T-cell receptor-modified T cells. The FDA goes on to state that, so as to get considerations particular to these, highly specialized sponsors have to communicate with the Office of Tissues and Advanced Therapies-OTAT in the Center for Biologics Evaluation and Research- CBER prior to the submission of an investigational new drug application.
Especially, the guidance goes on to discuss CAR-T cell design as well as development, including vectors, CAR constructs, cellular starting material, along with fresh or cryopreserved final products. Vector manufacturing, along with testing, as well as collection, handling, and testing of cellular starting material, happen to be addressed. Managing manufacturing changes along with assessing comparability across the CAR T-cell product lifecycle and single-site or multi-site CAR T cell manufacturing also happen to be discussed within the guidance document.