Tag: betibeglogene autotemcel

ICER To Look at Gene Therapy for Thalassemia

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 […]

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A Majority of Patients Achieve Transfusion Independence in Phase 3 Trials of LentiGlobin

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 […]

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