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STAT Plus: Europe approves first gene therapy to treat rare blood disease

A one-time gene therapy from Bluebird Bio won approval in Europe on Monday, marking a major milestone and giving the company a chance to learn from the field’s commercial mistakes of the past.

Bluebird’s therapy, called Zynteglo, is now cleared to treat people with beta thalassemia, a rare, inherited disease that causes the production of impaired red blood cells and severe anemia. The treatment promises to fix the genetic cause of beta thalassemia and relieve patients from the blood transfusions they would otherwise need.

Continue to STAT Plus to read the full story…

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