The U.S. Food and Drug Administration (FDA) on Wednesday approved a gene therapy for a rare blood disease which is set to reach the market at a record $2.8 million for a single dose, according to a press release by the therapy’s creator, Bluebird Bio.
Beta-thalassemia is an inherited blood disorder that causes a patient’s blood to fail to circulate oxygen through the body, according to the FDA press release concerning the approval. Bluebird’s new therapy, Zynteglo, infuses patients with cells that have a working copy of the gene responsible for the disorder, allowing the patient to produce blood that functions properly, according to a Bluebird press release.
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