CDC Public Health Webinar Series on Blood Disorders

Emerging trends in gene therapy: Thalassemia as a case study


Event details

This special session on emerging opportunities in gene therapy is part of the Centers for Disease Control and Prevention (CDC) Public Health Webinar Series on Blood Disorders.

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. 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.

Presented by 

Stefano Rivella, PhD
Professor of Pediatrics, Children’s Hospital of Philadelphia (CHOP)
Scientific Director of The Sickle Cell and Red Cell Disorders Curative Therapy Center, CHOP

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

Moderated by

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

Learning objectives

  1. Describe the efficacy and safety of gene therapy approaches for hemoglobin disorders based on results from current clinical trials.
  2. Describe the differences between gene addition and gene editing in the approach to gene therapy for hemoglobin disorders.
  3. Describe how gene therapy may address unmet medical needs of people with thalassemia.


This webinar is free and open to health-care providers, pharmacists and public health researchers. Additional event details and a registration link are available on the Centers for Disease Control and Prevention website Blood Disorders Webinar - Save the Date | CDC