Gene Therapy: Bluebird’s Beti-Cel Sails Through US Panel Review On Strength of ‘Impressive’ Efficacy
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.
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Novartis is once again looking to be a pioneer in cell and gene therapy, though this time is selecting a potentially less complex administration method.
Outcomes and disease progression could be clear cut, lending the rare disease treatment to innovative contracting. But the amount of money at stake is likely to complicate things.
Committee endorses use for those who lack a matched donor for allogeneic hematopoietic stem cell transplant and says the treatment should be an option for patients with a matched, but unrelated, donor.