December 11, 2020 – CAF is pleased to share several of the presentations made at the 2020 CAF Online Mini Conference for Patients and Families, which took place virtually on Saturday, July 11. Click on the links below to watch recordings of the presentations. Overview: Novel Therapies in Thalassemia Medical Issues Related to COVID-19 for […]
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 […]
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 […]
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 […]
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 […]
December 6, 2020 – Patients with transfusion-dependent beta thalassemia who were treated in bluebird bio’s clinical trials with beti-cel (LentiGlobin) gene therapy have shown sustained efficacy for up to six years in follow-up analysis. These findings were presented by Dr. Janet Kwiatkowski of the Children’s Hospital of Philadelphia at the 2020 American Society of Hematology […]
December 1, 2020 – Today is #GivingTuesday, the International Day of Giving. Show that you care by making a special donation today to the Cooley’s Anemia Foundation and support all those living with #thalassemia. With fundraising efforts diminished by the Covid-19 pandemic, your donation this year is needed more than ever! Click on the link […]
