Upon completion, participants will learn about TX200-TR101, an investigational CAR-Treg cell therapy that uses patient's cells transduced with a lentiviral vector encoding for a CAR to recognize HLA-A*02 in a Phase 1/2 STEADFAST clinical study for the prevention of immunemediated rejection following HLA-A2 mismatched kidney transplantation from a living donor.
Upon completion, participants will learn about BIVV003, an autologous cell therapy that involves gene editing of a patient’s own hematopoietic stem cells (HSCs) using zinc finger nuclease (ZFN) technology to boost endogenous production of fetal hemoglobin (HbF) for treatment of sickle cell disease.
Upon completion, participants will be able to comprehend how in vitro biological assays are developed to determine potency of a cell therapy based on the product's mechanism of action and as per ICH guidelines.
Upon completion, participants will be able to understand the differences in the potency assay development for a genetically modified cell therapy product versus gene edited cell therapy product in addition to differences in potency assay requirement for a Phase 1/2 versus Phase 3 cell therapy product.
