Tag: gene therapy




FDA Approves bluebird bio’s ZYNTEGLO®, the First Gene Therapy for People with Transfusion-Dependent Beta-Thalassemia

zynteglo approved for beta-thalassemia

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.

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Gene Therapy Trial for ALD Halted

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 […]

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Gene Therapy and Other Advances Happen Because of You

February 25, 2013 – The fact that the first U.S.-based gene therapy trial for thalassemia has started (click here for details) is fantastic. Some might think it’s unbelievable we’ve progressed to this point. Actually, it’s fantastic but not unbelievable. We have reached this point because many members of the thalassemia community have dedicated years of […]

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Clinical Trials in Thalassemia Cell and Gene Therapy Grants

The Cooley’s Anemia Foundation invites national and international applicants to apply for grants to facilitate clinical trials in Cell and Gene Therapy to advance a cure for thalassemia.   The purpose of this initiative is to speed the application of recent translational advances in cell and gene therapy to clinical trials. Both phase I (safety) and […]

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