Stefano Rivella, Ph.D.

Stefano Rivella, PhD, the Kwame Ohene-Frempong endowed chair in Pediatric Hematology and scientific director of the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders at Children’s Hospital of Philadelphia, is an expert in the use of lentiviral vectors for modulation of gene expression and gene transfer into hematopoietic stem cells and for the cure of hematological disorders. More recently, he expanded his focus on developing tools for RNA delivery (gene editing and RNA therapies) to treat hematological and metabolic disorders.

His research program generated the first adult mouse models of a- and b-thalassemia major and contributed to the creation of the first lentiviral vectors that corrected the hemoglobin synthesis in mice affected by b-thalassemia. This led the way to gene therapy clinical trials for b-thalassemia and sickle cell disease. An advanced vector generated in Dr. Rivella’s Lab for the cure of both b-thalassemia and sickle cell disease has been recently approved by the U.S. Food and Drug Administration for a clinical trial that will start at CHOP.

The Rivella Lab also is studying the role of seminal factors contributing to pathophysiology of hematopoiesis-, inflammation-, and iron-related disorders, such as dyserythropoietic anemias, polycythemia vera, anemia of inflammation, and hemochromatosis, to name a few. Based on some of these studies, the lab is developing or contributing to the characterization of novel therapeutics, while several pharmaceutical companies are currently testing new drugs based on his discoveries.