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Gene Therapy: Bluebird’s Beti-Cel Sails Through US Panel Review On Strength of ‘Impressive’ Efficacy

 
• By Sue Sutter

Treatment is a potential game-changer for patients with transfusion-dependent beta-thalassemia, committee members said; panel was hesitant to extrapolate the hematological malignancies seen with bluebird’s other lentiviral vector programs to beti-cel.  

Sailboat
Bluebird found smooth sailing for its beta-thalassemia gene therapy at a US FDA meeting. • Source: Shutterstock

Bluebird bio’s betibeglogene autotemcel (beti-cel) sailed through a US Food and Drug Administration advisory committee review on 10 June, with the agency’s external advisers saying the gene therapy’s “impressive efficacy” and “minimal risk” make it a potential game-changer in the treatment of transfusion-dependent beta-thalassemia.

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