Put these upcoming informational webinars on your calendar!
bluebird bio, Inc. announced the FDA has approved ZYNTEGLO®, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell transfusions.
The Institute for Clinical and Economic Review (ICER) announced today that it will assess the comparative clinical effectiveness and value of betibeglogene autotemcel (Zynteglo/LentiGlobin, bluebird bio) for the treatment of beta thalassemia. An FDA decision on betibeglogene autotemcel is expected in mid-2022. ICER is inviting public comment on its initial Draft Scoping Document, which details […]
August 9, 2021 – bluebird bio has announced that a patient in their gene therapy trial for early cerebral adrenoleukodystrophy (ALD) has developed MDS (myelodysplastic syndrome). In line with their protocols, bluebird has shared relevant data and sought advice about this report with the regulatory authorities, trial investigators, and the independent Data Monitoring Committee for […]
June 12, 2020 – Bluebird bio has reported the findings from its Phase 3 clinical trial program of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia). These show that pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia are able to achieve and maintain transfusion independence with hemoglobin (Hgb) levels that are near-normal (≥10.5 […]