TRANSLATIONAL CENTER OF EXCELLENCE FOR HEMATOPOIETIC STEM CELL ENGINEERING

The University of Pennsylvania Perelman School of Medicine (PSOM), in collaboration with the Division of Hematology-Oncology in the Department of Medicine and the Abramson Cancer Center (ACC) are thrilled to house a new Translational Center of Excellence (TCE) in Genetically Engineered Hematopoietic Stem Cell Transplantation.

The mission of this TCE is to catalyze translational research in gene-engineered hematopoietic stem cell therapy and to drive first-in-human testing through the ACC's Cell Therapy Trials unit.

A new era of genetically engineered hematopoietic stem cell products has emerged to treat sickle cell disease, thalassemia and other non-malignant indications. In cancer therapeutics, genetic engineering has been used for modification of immune effector cells such as T cells, including chimeric antigen receptor (CAR) T cells. While CAR T cells have made a major impact in lymphoma and myeloma, their impact in other cancers has been limited owing to lower than expected antitumor efficacy and high rates of toxicity arising from the lack of cancer-specific targets. This TCE will establish new approaches to gene editing of hematopoietic stem cells in order to shift the paradigms and boundaries of cutting-edge cell therapy.