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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