Sangamo Therapeutics and Bioverativ Therapeutics Begin New Clinical Trial for Transfusion-Dependent Beta-Thalassemia
April 6, 2018 – Sangamo Therapeutics and Bioverativ Therapeutics have begun a Phase 1/2 study of gene editing in patients with transfusion-dependent beta-thalassemia in the United States. This trial will investigate whether increasing the production of fetal hemoglobin can reduce or eliminate the need for blood transfusions.
Participating patients will have their CD34+ blood stem cells removed and genetically edited so as to boost their production of fetal hemoglobin. These edited stem cells will then be infused back into patients. The trial will enroll 6 patients between the ages of 18 and 40 with transfusion-dependent beta thalassemia. The first trial site has just opened in Oakland and additional trial sites are expected to open soon. Additional information is available at: http://bit.ly/SangamoBioverativeTrial