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 patients will have their CD34+ blood stem cells removed and genetically edited using CRISPR/Cas9 gene editing technology so as to reactivate their production of fetal hemoglobin. These edited stem cells will then be infused back into patients.

The trial will initially enroll 12 adult patients between 18 and 35 years of age with transfusion-dependent beta thalassemia, and may later be expanded to include up to 45 patients. Eligible patients include those with homozygous β-thalassemia (with the exception of the β0/β0 genotype) or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE), with a history of ≥10 units/year of packed RBC transfusions in the prior 2 years. This trial has been enrolling patients in Europe and in Canada and just reported positive results for the first treated patient, who is now transfusion-independent.

The first U.S. trial site has just opened in Nashville, TN at The Children’s Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers. Further information about the study is available at:

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