Topic: Current and Future Strategies for Gene Therapy

Dr. Rivella is Professor of Pediatrics at the Children’s Hospital of Philadelphia (CHoP), University of Pennsylvania, holds the Kwame Ohene-Frempong Chair on Sickle Cell Anemia and he is the Scientific director of the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED) at CHOP. He is also a member of the Institute for Regenerative Medicine, University of Pennsylvania; of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics-CHOP; Penn Center for Musculoskeletal Disorders; Gene Therapy and Vaccine program (GTV) of the Cell and Molecular Biology Graduate Group (CAMB), and Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia.

Dr. Rivella is an expert in the use of lentiviral vectors for modulation of gene expression and gene transfer into hematopoietic stem cells and 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 alpha- and beta-thalassemia major and contributed to the creation of the first lentiviral vectors that corrected the hemoglobin synthesis in mice affected by beta-thalassemia. This led the way to gene therapy clinical trials for b-thalassemia and sickle cell disease. An advanced vector generated in Dr. Rivella lab for the cure of beta-thalassemia and sickle cell disease has been recently approved by the FDA for a clinical trial starting at CHOP.

Dr. Rivella is also working on generating novel in vivo gene delivery tools (“In vivo modification of hematopoietic stem cells by targeted lipid nanoparticles delivering mRNA.” Science, July 2023) for the treatment of a variety of diseases.

The Rivella Lab also is studying the role of seminal factors contributing to the pathophysiology of hematopoiesis-, inflammation-, and iron-related disorders, such as dyserythropoietic anemias, polycythemia vera, anemia of inflammation, and hemochromatosis, to name a few. He characterized the role of seminal factors contributing to the morbidity and mortality in beta-thalassemia, sickle cell anemia, Polycythemia vera, hemochromatosis, and in anemia of inflammation, such as hepcidin, interleukin-6, and ferroportin. Based on some of these studies, the lab is developing or contributing to the characterization of novel therapeutics, while several pharmaceutical companies are presently testing new drugs based on his discoveries.

Dr. Rivella is presently an active member of the International BioIron Society (IBIS), American Society of Hematology (ASH), European Hematology Association (EHA), and American Society of Gene and Cell Therapy (ASGCT). For his research, Dr. Rivella has been awarded the New Investigator Award at the International Society of Experimental Hematology, the Sultan bin Khalifa International Thalassemia Federation Award, the Marcel Simon Award from the International BioIron Society (IBIS), numerous travel and fellowship awards from several international scientific societies, grants from the Cooley’s Anemia Foundation, Roche Foundation for Anemia Research (RoFAR) and 8 grants from the National Institutes of Health (NIH) as a PI.

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