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 […]
The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassemia. The awards are in three categories: Clinical Trials in Thalassemia Cell & Gene Therapy Clinical Research Application Research Fellowship Application Details are as follows: CLINICAL TRIALS IN THALASSEMIA CELL AND GENE THERAPY The Cooley’s Anemia Foundation invites […]
The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassemia.
Are you or a loved one considering gene therapy as a treatment option for transfusion dependent beta thalassemia? Watch the recording of the Gene Therapy Webinar featuring Dr. Sujit Sheth and reps from bluebird bio now!
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.
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 […]
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 […]
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 […]
