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Expert Interview
Slingshot members are talking to an expert! The topic is:
A Second View: Diving into the EDIT-301 ASH Clinical Data Abstract and the approval of CASGEVY & Lyfgenia
Ticker(s): EDIT, BLUE, CRSP, VRTX, BBIOWho's the expert?
Institution: CHOP
- 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.
- Very familiar with NORTHSTAR data on Zynteglo
- Dr. Rivella has a long-term expertise in the pathophysiology and genetics of several murine models of hematopoiesis-, inflammation-, and iron-related disorders as well as in the use of lentiviral vectors for modulation of gene expression and gene transfer for the cure of hemoglobinopathies. Dr. Rivella 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 such hepcidin, intereleukin-6 and ferroportin, the phosphokinase Jak2 and macrophages.
Interview GoalThis interview aims to explore the results and clinical insights garnered from the Phase I/II RUBY and EdiThal studies evaluating EDIT-301, a novel gene-edited autologous hematopoietic stem cell therapy, in patients with severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). We will focus on understanding the safety, efficacy, and implications of this innovative approach for managing these hereditary blood disorders. We will also discuss the recent approvals from Bluebird Bio and Crispr Therapeutics.
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