Pediatric Patients Now Eligible to Enroll in Clinical Trial of CTX001

September 2, 2020 – 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. This trial was initially enrolling adult patients only. However, it is now enrolling pediatric patients ages 12 and older. Patients between 12 and 35 years of age with transfusion-dependent beta thalassemia – including those with homozygous β-thalassemia (with the exception of the β0/β0 genotype) or hemoglobin E (HbE), with a history of ≥10 units/year of packed RBC transfusions in the prior 2 years- are eligible to enroll. This trial has recently reported positive results for the first two treated patients, who are now transfusion-independent. Patients may enroll at the following 3 U.S. trial sites: (1) The Children’s Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers in Nashville, TN; (2) Columbia University in NYC; and (3) Stanford University.

Further information about the study is available at:

Further information about the recent positive trial results can be found at:

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