New Bluebird Bio Data Shows Gene Therapy Promising for Thalessemia

Rare Daily Staff

Bluebird Bio said data from its phase 1/2 Northstar study of its experimental LentiGlobin gene therapy in adolescents and adults with transfusion-dependent beta-thalassemia and its ongoing phase 3 Northstar 2 study suggest a one-time treatment may address the underlying cause of the condition.

Transfusion-dependent beta-thalassemia is a severe genetic disease characterized by reduced or absent hemoglobin levels that results in severe anemia and ineffective red blood cell production. People with condition face a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.

Despite the availability of supportive care, many people with transfusion-dependent beta-thalassemia experience serious complications and organ damage due to underlying disease and iron overload.

The company said that the majority of patients with transfusion-dependent beta-thalassemia and non-beta/beta genotypes treated with its gene therapy are transfusion-free and producing total hemoglobin at normal or near-normal levels. The late-stage study used a refined manufacturing approach of the gene therapy intended to improve its results. Data from the studies will be presented June 16 at the 3^rd Annual Congress of the European Hematology Association.

Eight of ten non-beta/beta genotypes in the phase 1/2 study are transfusion-free for a median of 33 months. Seven of eight patients in the phase 3 study with at least six months follow-up are transfusion free for 4.7 to 15.1 months.

“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-beta/beta genotypes,” said Franco Locatelli, lead investigator of the company’s late-stage LentiGlobin study. “These results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.”

The company said it is on track to submit a marketing authorization application in the European Union later this year.

June 15, 2018
Franco Locatelli, lead investigator of Bluebird Bio’s late-stage LentiGlobin study