Common Questions

Answers to frequently asked questions about beta-thalassemia, gene therapy and clinical research studies are below.  
The studies are sponsored by bluebird bio, a Cambridge, Massachusetts-based biotechnology company dedicated to developing innovative gene therapies for severe genetic disorders. bluebird bio has several clinical programs in development for beta-thalassemia major, cerebral adrenoleukodystrophy and sickle cell disease. At the heart of bluebird bio’s efforts is the development of investigational gene therapy treatments for severe diseases with few or no clinical options.
You may sign up to receive updates on future gene therapy studies in people with beta-thalassemia.
bluebird bio presented updated data on the Northstar (HGB-204) Study at the Annual Meeting of the American Society of Hematology in December 2016. Early data from the Northstar Study shows that patients with non-β00 genotypes and ≥12 months of follow-up have stopped regular transfusions; patients with β00 genotypes and ≥12 months of follow-up had a median reduction in transfusion volume of 63%.
The best time to share your experience is after the study is finished. When patients speak publicly about their clinical study experience, their words may have unintended impact, even subconsciously, on how other study participants or even investigators report the ongoing results of the clinical study.
As the sponsor of the Northstar Studies clinical study, bluebird bio respects the privacy of the patients participating in our clinical studies and the personal healthcare data generated over the course of the study. Only the medical personnel associated with the study will have access to your medical information. If information from results of the Northstar Studies is published in the future, your personal information, such as your name, will not be included.
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The same lentivirus, known as LentiGlobin® BB305, was previously used in the Northstar Study, which enrolled adults who are transfusion-dependent and diagnosed with beta-thalassemia major. Early data from the Northstar Study shows that patients with non-β00 genotypes and ≥12 months of follow-up have stopped regular transfusions; patients with β00 genotypes and ≥12 months of follow-up had a median reduction in transfusion volume of 63%. Prior to the Northstar Study, three patients were treated in a gene therapy clinical study in France using a similar lentiviral vector between 2006 and 2011. None of the patients have experienced serious, unexpected side effects. However, not all short and long term risks are known at this time, and the patients continue to be followed by their physicians.
A genotype is an individual’s collection of genes. The genotype of a particular gene, like the gene for beta-globin, refers to the particular mutation a patient has in that gene. For a disease like transfusion-dependent beta-thalassemia, there are many different mutations (dozens or even hundreds) which can cause problems in the beta-globin gene. An individual patient with beta-thalassemia will only have one or two mutations in the beta-globin gene — these mutations are their genotype.
The Northstar Study is fully enrolled. The Northstar-2 Study is seeking 15 people, ≤50 years old, living with transfusion-dependent beta-thalassemia who cannot make enough of the beta-globin portion of hemoglobin and who do not have the β00 genotype.
The purpose of the studies is to determine if the one-time gene therapy can decrease or eliminate the need for continued blood transfusions, while also being safe and well-tolerated.
The goal of this investigational gene therapy treatment is to help people living with transfusion-dependent beta-thalassemia make more beta-globin and produce healthy red blood cells.
Gene therapy uses functioning genes as medicine for people who have a gene that is malfunctioning.