Tag: pharma

Thalassemia Care Walk 2024 – Information

Care Walk is the Cooley’s Anemia Foundation’s annual fundraising event and opportunity to bring together the thalassemia community and its supporters around the country. Funds raised support medical research to fight thalassemia, a genetic blood disorder, as well as patient support services for thalassemia patients across the country. Click here to register for Care Walk!! […]

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FDA Approves Vertex & CRISPR’s Gene Editing Therapy to treat Beta Thalassemia

Treat Beta Thalassemia

The FDA has approved Vertex and CRISPR’s gene-editing therapy Casgevy to treat beta thalassemia. Casgevy becomes the first therapy for the rare blood disorder to use the innovative CRISPR gene-editing technology. Vertex Pharmaceuticals and CRISPR Therapeutics have scored an FDA approval for their gene-editing therapy Casgevy (exa-cel) to treat transfusion-dependent beta thalassemia (TDT). The approval […]

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Agios Pharmaceuticals Announces Phase 3 ENERGIZE – Treatment for Thalassemia Patients

Phase 3 ENERGIZE

Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia January 3, 2024 – Agios Pharmaceuticals, Inc. today announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of […]

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Agios Launches Advocacy Advisory Council for Thalassemia, PK Deficiency, SCD

The council will bring together patients, caregivers and physicians from these communities, which share commonalities in underlying disease pathology, difficulties in transitioning from pediatric to adult care and quality of life concerns.

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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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Agios Pharmaceuticals’ Oral Mitapivat Increases Hemoglobin Levels and Improves Markers of Erythropoiesis in Patients with Non-Transfusion-Dependent Thalassemia

December 7, 2020 – Agios Pharmaceuticals reported preliminary findings from their Phase 2 study of mitapivat in non-transfusion-dependent thalassemia at the 2020 American Society of Hematology (ASH) virtual medical conference. Mitapivat activates the pyruvate kinase-R (PKR) enzyme, which improves red blood cell energy metabolism. Mitapivat was shown to improve ineffective erythropoiesis and anemia in a […]

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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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