Author: Craig

ASH Report: Luspatercept and Iron

December 6, 2020 – The effect of long-term luspatercept use on iron levels and on utilization of iron chelation therapy was assessed in the Phase 3 BELIEVE trial of luspatercept in adult patients with transfusion-dependent beta thalassemia. After 24 weeks of treatment, 17.0% of patients who took luspatercept showed a reduction in serum ferritin levels […]

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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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ASH Report: Pediatric Results in bluebird bio Gene Therapy Trial

December 6, 2020 – Initial results from Phase 3 clinical trials showed that 10/12 adult patients with transfusion-dependent beta thalassemia achieved transfusion independence following treatment with bluebird bio’s beti-cel (LentiGlobin) gene therapy for β-thalassemia. After showing initial success in adult patients, these trials were expanded to include adolescents and children. Preliminary findings from the pediatric […]

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ZYNTEGLO Gene Therapy Launches in Germany for Treatment of Beta Thalassemia

January 27, 2020 – Earlier this month, bluebird bio announced the launch in Germany of ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene), the first gene therapy approved in Europe for the treatment of transfusion-dependent beta thalassemia. ZYNTEGLO is a one-time gene therapy for patients 12 and older with transfusion-dependent beta-thalassemia who do nothave a B0/B0 […]

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Presentations from 2019 CAF Patient-Family Conference

January 23, 2020 – CAF is pleased to share several of the presentations made at the 2019 CAF Patient-Family Conference, which took place from July 12-14 in San Francisco, CA. Click on the links below to download the presentations. Fetal Therapy for Alpha and Beta Thalassemia Major – Tippi Mackenzie, MD Issues in Health Insurance: […]

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First Trial Site Opens in Nashville for CRISPR/VERTEX Gene Editing Therapy for Beta Thalassemia

November 20, 2019 – CRISPR Therapeutics and Vertex are conducting a Phase 1/2 study of gene editing in patients with transfusion-dependent beta-thalassemia. This trial will investigate whether increasing the production of fetal hemoglobin through gene editing can reduce or eliminate the need for blood transfusions. It will also investigate the safety of this treatment. Participating […]

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