Tag: pharma



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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New CAF-Acceleron Scholarships for Academic Achievements Accepting Applications from U.S. Thalassemia Community

March 31, 2021 – The Cooley’s Anemia Foundation and Acceleron Pharma Inc. are offering up to four college scholarships of $5,000 each to individuals with a clinically significant form of thalassemia (alpha or beta thalassemia major, beta thalassemia intermedia, e beta thalassemia, hemoglobin H disease or hemoglobin H disease Constant Spring).  Two of these scholarships […]

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ASH Report: Early Results from CTX001 Gene Editing Study

December 6, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal […]

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Two Patients Become Transfusion Independent After Gene Editing Therapy

June 12, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal hemoglobin […]

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FDA Approves Twice-Daily Form of Ferriprox

A new formulation of the oral chelator Ferriprox, which is intended to be taken twice a day, has been approved for use in the United States by the U.S. Food and Drug Administration.  The previously approved formulation was to be taken three times daily; the new formulation eliminates the mid-day dose. “In the management of patients […]

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