Visiting Scholars

Stefano Rivella, Ph.D.

Visiting Faculty / Professor, CHoP

Dr. Rivella is Professor of Pediatrics at the Children’s Hospital of Philadelphia (CHoP), holds the Kwame Ohene-Frempong Chair on Sickle Cell Anemia, is the Scientific director of the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED) and leader of the RNA Gene Therapeutics of the Penn Institute for RNA Innovation (led by Dr. Drew Weissman).  Through his translational efforts, Dr. Rivella has helped to bring life-changing medical advancements to patients. He contributed to the development of the first lentiviral vector to cure beta-thalassemia, paving the way for current gene complementation technologies. He generated the first murine models of adult beta- and alpha-thalassemia major, XLSA and CDA1 (sideroblastic and dyserythropoietic anemias), metachromatic leukodystrophy , and related curative gene therapy approaches.  He was involved in the characterization of Luspatercept-Reblozyl and was the first to propose the use of hepcidin agonists/inducers in beta-thalassemia, hemochromatosis, and polycythemia vera. For instance, Luspatercept has been approved to improve red cell production in beta-thalassemia. At the same time, a hepcidin agonist (Rusfertide) has shown its potential to replace phlebotomy without causing iron deficiency in polycythemia vera patients. Dr. Rivella is currently supporting a clinical trial using a lentiviral vector, generated in his laboratory, to cure hemoglobinopathies. He expanded his focus on developing tools for RNA delivery (gene editing and RNA therapies) to treat hematological and metabolic disorders. He also uses this technology to target hematopoietic stem cells in vivo as an alternative, non-toxic approach to myeloablation.