Early Data for bluebird bio Gene-Therapy Candidate Shows Some Promise

06:21 EDT 23 Jun 2017 | Genetic Engineering News

bluebird bio today disclosed its first data, including some promising results, from a pair of clinical studies assessing its LentiGlobin™ gene therapy candidate—including a Phase III study that followed a change in its manufacturing process. In the Phase III Northstar-2 (HGB-207) study of LentiGlobin in patients with transfusion-dependent β-thalassemia (TDT) and non-β 0 /β 0 genotypes, early interim data showed that the first 3 of 15 patients treated to date achieved higher drug product vector copy number (DP VCN) and lentiviral vector positive (LVV+) cell production than in the earlier Northstar (HGB-204) study, bluebird said. Those results are correlated with higher production of hemoglobin A (HbA) T87Q and ultimately may address known patient-to-patient variability, bluebird bio CMO David Davidson, M.D., said in a statement. “The first patient treated in this study exemplifies the promise of gene therapy: discontinuing blood transfusions approximately a month after treatment and ...

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