A Second View: Revolutionizing Sickle Cell Disease and Thalassemia Treatment: A Deep Dive into the EDIT-301 ASH Clinical Data Abstract and the approval of CASGEVY & Lyfgenia

Executed on:Dec 20, 2023 4:00 PM EDT

Call Duration:30 minutes

Expert Profile

ExpertBlinded Expert

Institution: Rutgers Medical School

Clinical Section Chief, Pediatric Hematology/Oncology and Director, Comprehensive Sickle Cell Center at Rutgers Cancer Institute of New Jersey; Vice Chair, IRB at Rutgers Biomedical and Health Sciences; and Vice Chair, NCI Pediatric Central IRB.
Currently manages 450 patients with sickle cell disease and sees 20/year with cisplatin induced hearing loss.
Research focuses on Hodgkin Disease, Pediatric oncology/ hematology, and Sickle cell disease.

Interview Goal

This 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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