Gene therapy is a transformative, and potentially curative, treatment option for patients with sickle cell disease (SCD). There are currently two gene therapies approved by the U.S. Food and Drug Administration (FDA) for the treatment of SCD in patients aged 12 years or older: lovotibeglogene autotemcel (Lyfgenia, bluebird bio), a lentiviral gene therapy; and exagemglogene autotemcel (Casgevy, Vertex), a CRISPR-Cas9 gene editing therapy.1
“Gene therapy has greatly expanded our treatment options for patients with SCD,” said Robert Sheppard Nickel, MD, MSc, associate professor in the divisions of hematology and blood and marrow transplantation at Children’s National Hospital in Washington, D.C. “Although we have had allogeneic bone marrow transplant for more than 20 years, there are a lot of issues with that approach—the biggest one being that a lot of patients don’t have a matched donor. Gene therapy offers another curative therapy to patients that don’t have good donor options.”
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September 2025
Transfusion is AABB’s scholarly, peer-reviewed monthly journal, publishing the latest on technological advances, clinical research and controversial issues related to transfusion medicine, blood banking, biotherapies and tissue transplantation. Access of Transfusion is free to all AABB members.
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