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Gene Therapy
Bluebird scores second gene therapy approval
by Shi En Kim
September 25, 2022
| A version of this story appeared in
Volume 100, Issue 34
Bluebird Bio has received approval from the US Food and Drug Administration for the gene therapy elivaldogene autotemcel, branded as Skysona. The treatment slows the progression of neurologic dysfunction in young boys with cerebral adrenoleukodystrophy. The go-ahead comes within a month of an FDA approval for Bluebird’s other gene therapy, Zynteglo, which treats the blood disorder β-thalassemia. Priced at $3 million for a full treatment, Skysona has eclipsed Zynteglo as the world’s most expensive drug.
Chemical & Engineering News
ISSN 0009-2347
Copyright © 2024 American Chemical Society
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