LentiGlobin BB305 gene therapy: Additional Phase I/II data

Additional data from the first 2 patients with beta thalassemia major who received an autologous hematopoietic stem cell transplantation (HSCT) with LentiGlobin BB305 in the open-label, French Phase I/II HGB-205 trial showed that both patients remain transfusion independent at day 180 and day 90, respectively. The 2 patients received their last blood transfusion on post-transplant day 10 and 12, respectively. The first patient had a total hemoglobin level of 10.2 g/dL, of which 7.2 g/dL was generated by LentiGlobin BB305, at day 180 post-transplant and the second patient had a total hemoglobin level of 11 g/dL, of which 6.8 g/dL was generated by LentiGlobin BB305, at day 90 post-transplant. No serious or treatment-related adverse events were reported. The trial is designed to enroll up to 7 patients. Additional data will be presented at the American Society of Hematology meeting in San Francisco in December. bluebird bio previously reported that both patients achieved blood transfusion independence within 2 weeks of transplant (see BioCentury, June 23). ...