BioPharma Dive January 12, 2023
Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges.
Sometime before the end of March, a person with the inherited blood disease beta thalassemia will receive an infusion containing hundreds of millions of their own stem cells. These cells will have just completed a round-trip journey to a drug manufacturing facility, where they’ll be equipped with a modified gene capable of fixing the patient’s condition.
The expected infusion will be the first commercial use of a gene therapy approved by the Food and Drug Administration in August. Called Zynteglo, the treatment is the result of more than a decade of research by biotechnology company Bluebird bio. Its approval...
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