The Northstar Studies are global clinical studies being conducted to determine if the one-time gene therapy can decrease or eliminate the need for continued blood transfusions in people living with transfusion-dependent beta-thalassemia, while also being safe and well-tolerated.
Northstar-2 Study Now Enrolling
The study now enrolling, Northstar-2, is a Phase 3 global clinical study seeking to enroll patients who cannot make enough of the beta-globin portion of hemoglobin. See if someone you know may qualify.
Beta-thalassemia is an inherited blood disease that can cause severe anemia. Beta-thalassemia is one of the most common genetic diseases in the world, and approximately 60,000 children are born annually with the disease. Additional treatment options are needed.
Understanding Gene Therapy
Gene therapy uses functioning genes as medicine for people who have a gene that is malfunctioning. See how it works.