No one picked BLUE :-(
Bluebird Bio (BLUE) Stock Shot Up 43% After-Hours
Bluebird shares soared 43% in extended trading yesterday after the company reported encouraging trial results for its experimental gene therapy for patients with beta-thalassemia
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Bluebird bio Inc ( NASDAQ:BLUE) saw its share price explode in yesterday's extended trading session, rising by as much as 43% to reach $70 apiece, after the company announced that its experimental gene therapy, LentiGlobin BB305, had helped treat patients suffering from an inherited blood disorder without the required regular blood transfusions.
The Cambridge, Massachusetts-based clinical-stage biotech presented data from its trials at the 56th Annual Meeting of the American Society of Hematology held yesterday in San Francisco. Bluebird stated that four patients suffering from beta-thalassemia major went on to produce enough hemoglobin upon treatment with the company’s gene therapy, LentiGlobin BB305, to an extent where they no longer needed transfusions. The company further reported that one of the patients in the trials went on without a blood transfusion for as long as 12 months.
Gene therapy is a process of inserting genetically engineered genes into the patient’s own cells to correct an underlying genetic defect that causes abnormality in gene expression.
Beta-thalassemia is an inherited disease which causes severe anemia, rendering the patients unable to produce the beta-globin part of the hemoglobin – the protein responsible for carrying oxygen in the body. The company estimates that about 40,000 children are born with beta-thalassemia, making it one of the most common inherited diseases. Beta-thalassemia, if left untreated, can prove to be fatal. Traditional forms of treatment for the disease include regular blood transfusions; however, the process itself can cause excess iron buildup in the body, which can damage tissues and cause cirrhosis. LentiGlobin BB305, on the other hand, is a one-time therapy involving the removal of the patient’s hematopoietic stem cells, which are then genetically modified and then re-inserted in the body. So far, there have been no serious side-effects reported from the procedure.
Bluebird has plans to talk to the US and EU regulatory bodies in an attempt to accelerate the development of the said drug. The company plans to file for an accelerated review process that will allow it to begin working on a late-stage study instead of waiting for 24-month data. |
