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FDA approval
Bluebird Bio’s sickle-cell gene therapy receives FDA approval
Cell & Gene Therapy
Dec 8, 2023
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Cell & Gene Therapy

Cell & Gene Therapy

Dec 8, 2023

Bluebird Bio’s sickle-cell gene therapy receives FDA approval

FDA approval

  • Gene therapy company Bluebird Bio has received FDA approval for its one-time gene therapy, LYFGENIA, for the treatment of sickle-cell disease in patients 12 years and older who have experienced vaso-occlusive events (VOEs). The drug, which is priced at USD 3.1 million, will be available in the market in early 2024.

  • LYFGENIA helps treat sickle-cell disease by integrating a functional β-globin gene into patients' blood stem cells. This process potentially enables sustainable production of adult hemoglobin with anti-sickling properties, reducing VOEs, and blood vessel blockage, causing tissue damage and pain. It is a notably custom-designed therapy to treat the root cause of sickle-cell disease. 

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