Company Update (NASDAQ:BLUE): bluebird bio Inc Opens Phase 3 Study of LentiGlobin™ Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia

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 the opening of HGB-207, a Phase 3, global, multi-center study in patients with transfusion-dependent beta-thalassemia with non-β00 genotypes. This study will incorporate the addition of bluebird bio’s transduction enhancers to the hematopoietic stem cell (HSC) manufacturing process, and will be conducted under the same Investigational New Drug application (IND) as previous studies of LentiGlobin in beta-thalassemia.

“The opening of bluebird’s first Phase 3 study for LentiGlobin, in which we are using an improved manufacturing process that increases transduction efficiency, is an exciting step forward,” saidDavid Davidson, M.D., chief medical officer, bluebird. “The accumulating clinical data show a correlation among vector copy number, the percentage of vector-containing cells, and the amount of hemoglobin produced by patients treated with LentiGlobin. We believe that the addition of our transduction enhancers to our manufacturing process has the potential to substantially increase the hemoglobin levels in patients with transfusion dependent beta-thalassemia and increase their likelihood of achieving clinically meaningful reductions in transfusion requirements or transfusion independence, the ultimate goal of our therapy.”

In this study, the process by which the patient’s cells are transduced with LentiGlobin will be modified by the addition of two additives during the transduction step of the manufacturing process, intended to increase vector copy number and the percentage of cells successfully transduced. bluebird also intends to incorporate these transduction enhancers into the manufacturing process for HGB-206, its ongoing study of LentiGlobin in patients with severe sickle cell disease (SCD). (Original Source)

Shares of Bluebird bio closed yesterday at $52.81, up $2.16 or 4.26%. BLUE has a 1-year high of $143.08 and a 1-year low of $35.37. The stock’s 50-day moving average is $53.50 and its 200-day moving average is $47.08.

On the ratings front, Bluebird has been the subject of a number of recent research reports. In a report issued on August 22, Jefferies Co. analyst Brian Abrahams reiterated a Buy rating on BLUE. Separately, on August 18, Goldman Sachs’ Salveen Richter maintained a Buy rating on the stock and has a price target of $135.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Brian Abrahams and Salveen Richter have a total average return of 5.4% and 30.9% respectively. Abrahams has a success rate of 55% and is ranked #533 out of 4147 analysts, while Richter has a success rate of 68.5% and is ranked #84.

Overall, one research analyst has assigned a Hold rating and 6 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $91.33 which is 72.9% above where the stock closed yesterday.

bluebird bio, Inc. is a clinical-stage biotechnology company. The company develops next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. It has two clinical-stage programs in development for childhood cerebral adrenoleukodystrophy and beta-thalassemia/sickle cell disease and a preclinical oncology program in the chimeric antigen receptor T cells field. 


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