Product Pipeline
Beta-Thalassemia and Sickle Cell Anemia
Product Development
bluebird bio’s LentiGlobin® introduces a fully functional human beta-globin gene, under the control of the beta-globin promoter and locus control regions, into the patient's own hematopoietic stem cells. bluebird bio is conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell anemia. By adding a single transduction step to the routine practice of autologous bone marrow transplantation, LentiGlobin may extend the benefit of bone marrow transplantation to the 75 to 85 percent of patients lacking matched sibling donors. LentiGlobin therapy would avoid the mortality and morbidity associated with graft-versus-host disease and immune suppression. It could also eliminate the need for monthly transfusions and daily overnight intravenous iron chelation therapy to fight the dangerous overload of iron that is caused by chronic transfusions.
About Beta-Thalassemia and Sickle Cell Anemia
bluebird bio is conducting Phase 1/2 trials with its LentiGlobin product in beta-thalassemia/sickle cell anemia, the two most prevalent human genetic disorders.
Thalassemias are inherited blood disorders that cause the body to have an inadequate amount of hemoglobin, the protein in red blood cells that carries oxygen. Beta-thalassemia is named for defects in production of the beta-globin chain of hemoglobin and mostly affects people of Mediterranean, Middle Eastern, South Asian, Southeast Asian and Chinese descent. Approximately 60,000 children are diagnosed with the disease each year throughout the world. The severe form of beta-thalassemia, also known as thalassemia major or Cooley's anemia, occurs when both beta-globin genes (one from each parent) are mutated. In beta E /beta 0-thalassemia one of the mutated genes is completely silent while the other expresses low levels of a mutated protein (beta E). This form is especially common in Southeast Asian countries and their diasporas. Immigration patterns have made this form the most frequent in the West Coast of the United States. Patients typically require monthly supportive red blood cell transfusions to treat their severe anemia for life. Unfortunately, over time these transfusions lead to severe iron overload that causes serious damage to many organs. Patients must regularly take chelating drugs to remove the excess iron to stay alive. The other option – allogeneic stem cell transplantation – carries a significant risk of morbidity and mortality.
Sickle cell anemia is characterized by clotting of improperly shaped red blood cells, which leads to a wide variety of serious health problems including chronic pain and high risk for stroke.
