Cure for Genetic Beta Thalassemia Nears Discovery

UCSF Benioff Children's Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe anemia, with a need for lifelong blood transfusions and the risk of fatal organ damage.

The Oakland hospital was one of three sites in the United States to host the trials for the Bluebird Bio therapy, which the U.S. Food and Drug Administration approved in August 2022. UCSF Benioff Oakland is now one of only 15 medical centers certified to provide the gene therapy Zynteglo and is among the first to treat a patient.

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