Description
Zynteglo (betibeglogene autotemcel) is a gene therapy used to treat beta-thalassemia, a rare inherited blood disorder that causes reduced production of hemoglobin and leads to severe anemia.
It works by inserting a functional copy of the beta-globin gene (HBB) into the patient’s own stem cells, enabling the body to produce normal hemoglobin and reduce or eliminate the need for regular blood transfusions.
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