LentiGlobin BB305 gene therapy: Phase I/II data

bluebird bio said the first patient with beta thalassemia major who received an autologous hematopoietic stem cell transplantation (HSCT) with LentiGlobin BB305 in the open-label, international Phase I/II Northstar (HGB-204) trial experienced monthly increases in the proportion of therapeutic globin protein generated by LentiGlobin BB305 of 1.5% at month 1, 10.9% at month 2 and 19.5% at month 3. The patient had a total hemoglobin level of 8.6 g/dL, of which 1.77 g/dL was generated by LentiGlobin BB305 at month 3. No serious or treatment-related adverse events were reported. The trial is designed to enroll up to 15 patients. Additional data will be presented at the American Society of Hematology meeting in San Francisco in December. ...