General Session: Gene Therapy and Gene Editing as a Treatment Option for Patients with Hemoglobinopathies (103)

9:30 – 10:30 am Friday, September 4

Sickle cell disease is one of the most common inherited diseases in the Unites States, with a homozygous frequency of 1 in 400 African American births. The disease is associated with multiple complications including infections, veno-occlusive pain crisis, acute chest syndrome, and cerebrovascular accidents.

Although the survival of patients with sickle cell has improved over the decades, patients with the disease continue to experience significant complications. The disease is also associated with increased mortality, especially in adulthood. There are several therapies that can ameliorate the disease, including transfusion therapy, hydroxyurea, glutamic acid, and more recently, voxelotor. The only curative option for patients with sickle cell disease is allogeneic hematopoietic stem cell transplant. Unfortunately, less than 15% of patients have an HLA identical donor. Gene therapy and gene editing clinical trials use gene modified autologous stem cells that eliminate the need for a matched related donor. We will discuss the technology and treatment behind this approach. We will also present early results from the ongoing gene therapy and gene editing clinical trials.


Haydar Frangoul, MD MS


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