Public Health Webinar Series on Blood Disorders

Presenters

Stefano Rivella, PhD

Professor of Pediatrics, Children’s Hospital of Philadelphia
Scientific Director of The Sickle Cell and Red Cell Disorders Curative Therapy Center,
Children’s Hospital of Philadelphia

Sujit Sheth, MD

Chief, Division of Pediatric Hematology/Oncology
Professor of Clinical Pediatrics, Weill Cornell Medicine

Moderator

Muin J. Khoury MD, PhD
Director, Office of Genomics and Precision Public Health,
National Center on Birth Defects and Developmental Disabilities,
Centers for Disease Control and Prevention

Emerging Trends in Gene Therapy: Thalassemia as a Case Study — June 22, 2023, 2:00–3:00 PM ET

Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy.

In this presentation, Drs. Rivella and Sheth will review the status of the FDA-approved gene therapy process for thalassemia, discussing the science behind it, the results of the clinical trials associated with it, and the clinical implications as gene therapy begins to be applied in a real-world setting. In addition, they will explore future clinical trials involving potential alternative gene therapy treatments.