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Bluebird Bio says interim LengiGlobin data published in NEJM.

Bluebird Bio announced interim data published in the New England Journal of Medicine from two separate two-year clinical studies investigating the potential for LentiGlobin gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent beta-thalassemia. Both studies, Northstar, which recently was completed, and HGB-205, which is ongoing, are evaluating the safety and efficacy of one-time treatment with LentiGlobin gene therapy and the interim results showed that a majority of the 22 patients in the two Phase 1/2 studies followed for two years or longer remained free from transfusions. Interim results also showed that all but one patient with a non-beta0/beta0 genotype stopped receiving regular red blood cell transfusions, with a median time since last transfusion of 27 months. In the nine patients with a beta0/beta0 genotype or similar severity, median transfusion volume decreased by 73 percent, and RBC transfusions were stopped in three patients. Treatment with LentiGlobin requires an autologous stem cell transplant. The safety profile of LentiGlobin has been consistent with myeloablative conditioning with the chemotherapy agent busulfan. "These interim data demonstrate the potential of LentiGlobin gene therapy to address the underlying genetic cause of TDT and increase production of functional red blood cells," said Dave Davidson, M.D., chief medical officer, bluebird bio. "Nearly all patients in the two studies with a non-beta0/beta0 genotype achieved freedom from chronic blood transfusions and, importantly, several of these patients reached normal or near-normal total hemoglobin levels and sustained those levels throughout the interim study period. We hope the refined manufacturing process implemented in our ongoing pivotal trials of LentiGlobin will translate into further normalization of total hemoglobin levels across genotypes."

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Publication:The Fly
Date:Apr 18, 2018
Words:276
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