Interim data for LentiGlobin gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent beta-thalassemia is published in NEJM.- bluebird bio,Inc.

bluebird bio, Inc. announced interim data published in the New England Journal of Medicine (NEJM) from two separate two-year clinical studies investigating the potential for LentiGlobin gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent beta-thalassemia (TDT). Both studies, Northstar (HGB-204), which recently was completed, and HGB-205, which is ongoing, are evaluating the safety and efficacy of one-time treatment with LentiGlobin gene therapy and the interim results showed that a majority of the 22 patients in the two Phase 1/II studies followed for two years or longer remained free from transfusions. Interim results also showed that all but one patient with a non-beta0/beta0 genotype (12 of 13 patients) stopped receiving regular red blood cell (RBC) transfusions, with a median time since last transfusion of 27 months. In the nine patients with a beta0/beta0 genotype or similar severity, median transfusion volume decreased by 73 percent, and RBC transfusions were stopped in three patients. Treatment with LentiGlobin requires an autologous stem cell transplant. The safety profile of LentiGlobin has been consistent with myeloablative conditioning with the chemotherapy agent busulfan. transfusion-dependent thalassemia is a severe genetic disease characterized by reduced or absent hemoglobin production that results in severe anemia and ineffective red blood cell production. People with TDT need regular blood transfusions to survive, but chronic transfusions lead to unavoidable iron overload that can result in multi-organ damage and shortened life span.

�One-time treatment with LentiGlobin gene therapy resulted in positive outcomes for patients with TDT, with the majority of the 22 patients in the two Phase 1/II studies followed for two years or longer maintaining independence from transfusion without unexpected or unmanageable side effects,� said Dr. Alexis Thompson, Head of Hematology and Director of the Comprehensive Thalassemia Program at Ann & Robert H. Lurie Children�s Hospital of Chicago, and Professor of Pediatrics at Northwestern University Feinberg School of Medicine and one of the lead authors of the NEJM paper. �People with TDT cannot make enough hemoglobin in their red blood cells and rely on frequent transfusions to survive, which can cause serious complications. Most will not have a suitable donor for conventional allogeneic stem cell transplant. These results suggest that gene therapy could become an effective treatment for TDT.�

See- "Gene Therapy in Patients with Transfusion-Dependent beta-thalassemia"- Alexis A. Thompson, M.D., M.P.H., Mark C. Walters, M.D., Janet Kwiatkowski, M.D., John E.J. Rasko, M.B., B.S., Ph.D., Jean-Antoine Ribeil, M.D., Ph.D., Suradej Hongeng, M.D., Elisa Magrin, Ph.D., Gary J. Schiller, M.D., Emmanuel Payen, Ph.D., Michaela Semeraro, M.D., Ph.D., Despina Moshous, M.D., Ph.D., Francois Lefrere, M.D., et al.-April 19, 2018 N Engl J Med 2018; 378:1479-1493 DOI: 10.1056/NEJMoa1705342.