Bluebird Bio Presents Promising Results for Gene Therapy in Rare Blood Disorder

admin
admin
  • One time treatment may address underlying cause of the disease
  • Works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells
  • Evaluating LentiGlobin in both β-thalassemia and Sickle Cell Disease
  • LentiGlobin granted orphan drug status in both β-thalassemia and Sickle Cell Disease

Bluebird bio, Inc. (Nasdaq: BLUE) announced on 6/15/2018 that new data from the completed Phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent β-thalassemia (TDT) and any genotype, and its ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of LentiGlobin investigational gene therapy in patients with TDT and non-β0/β0 genotypes, will be presented in an oral session on June 16 at the 23rd Annual Congress of the European Hematology Association by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesù of Rome, Italy.

“The maturing data from HGB-204 and HGB-207 suggest that one-time treatment with LentiGlobin may address the underlying genetic cause of TDT. With our refined manufacturing process, the majority of patients with TDT and non-β0/β0 genotypes are transfusion-free and producing total hemoglobin at normal or near-normal levels,” said David Davidson, M.D., chief medical officer, bluebird bio. “We are on track to submit a marketing authorization application in the European Union later this year, and we continue to work closely with clinical investigators and regulatory authorities to complete our ongoing clinical trials and bring this important treatment option to patients as soon as possible.”

People with TDT need regular blood transfusions to survive, but chronic transfusions carry risks, including unavoidable iron overload that can result in multi-organ damage and shortened life expectancy. Eliminating or reducing the need for transfusions may reduce the long-term complications associated with TDT and current standards of care.


“Consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels in patients indicate that LentiGlobin manufacturing refinements have resulted in improved gene therapy characteristics and may enable sustained transfusion independence for a great majority of patients,” said Professor Locatelli, the lead investigator of the Northstar-2 study. “Further, we are now seeing more than three years of data from the Northstar study indicating that LentiGlobin therapy may enable long-term transfusion independence in the majority of patients with non-β0/β0 genotypes. These results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.

LentiGlobin works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells outside the body (ex vivo) and then transplanting those modified cells into the patient’s blood stream through infusion, also known as autologous stem cell transplantation.Bluebird bio is currently testing LentiGlobin in both sickle cell disease and β-thalassemia. The FDA has granted  LentiGlobin orphan drug status for both  β-thalassemia and sickle cell disease. Bluebird currently has four ongoing studies evaluating LentiGlobin in  β-thalassemia and sickle cell disease.