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Author Topic: Beta 0 Gene Therapy Trials to Begin Recruiting Patients  (Read 1679 times)
Andy Battaglia
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« on: November 17, 2017, 08:33:12 PM »

bluebird bio Announces First Patient Treated in Northstar-3 (HGB-212), Phase 3 Study of LentiGlobin™ in Patients with Transfusion-Dependent β-Thalassemia (TDT) and β0/β0 Genotype

– Study to enroll approximately fifteen adult, adolescent and pediatric patients –


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 16, 2017-- bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today announced that the first patient has been treated in Northstar-3 (HGB-212), the company’s Phase 3 study of LentiGlobin in patients with transfusion-dependent β-thalassemia (TDT) with the β0/β0 genotype.

“We are excited to start our second Phase 3 study of LentiGlobin in patients with TDT,” said Dave Davidson, chief medical officer. “Early data presented at the European Hematology Association annual meeting this year indicates that our refined manufacturing process, now implemented for treatment across all of our active studies of LentiGlobin, has consistently yielded improved Drug Products with higher vector copy number and an increased proportion of transduced cells. We are optimistic that these advances will improve our ability to achieve the levels of HbAT87Q needed for the patients in this study, who have the β0/β0 genotype, to see their annual RBC transfusions dramatically reduced, if not entirely eliminated.”

“Ongoing studies of LentiGlobin in patients with TDT have indicated the potential for a durable effect, with patients seeing consistent production of HbAT87Q for more than three years after treatment,” said Alexis Thompson, MD, MPH, Ann & Robert H. Lurie Children’s Hospital of Chicago, Illinois and a primary investigator on the study. “With the introduction of the refined manufacturing process into the Northstar-2 and Northstar-3 pivotal studies, we hope our patients will produce a greater amount of hemoglobin to enhance their treatment effect, and thereby demonstrate that LentiGlobin has the potential to address the underlying genetic cause of TDT, regardless of genotype.”


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