Shares of Bluebird Bio (BLUE) jumped 60% on the open after it had announced that one if its drugs, Lentiglobin BB305, which utilizes gene therapy, was able to allow patients to become transfusion free. Bluebird Bio is a biotechnology stock that is developing treatments for rare genetic disorders. This result of allowing patients to become tranfusion-free is major because these patients with beta-thalassemia major have a rare blood disorder that forces them to have to do chronic blood transfusions. These 5 patients treated with Lentiglobin BB305 come from two different studies. Two of the patients come from the NorthStar study ( HGB-204), and the other three patients come from the HGB-205 study. Lentiglobin is designed to treat beta-thalassemia major and quite possibly sickle cell disease. The way the drug works against beta-thalassemia major is that patient's cells are inserted with a modified version of a human-beta globin that allows them to produce beta-globin levels alone without the need to receive constant blood transfusions.
The patients receiving this gene therapy has allowed them to be transfusion-free for about 5 months now and is a huge breakthrough for this rare blood disorder. These results were presented at the 56th Annual Meeting of the American Society of Hematology — ASH. There are around 40,000 children born every year with a major form of this disease, and if left untreated, can be fatal. The reason being is that these patients are unable to produce enough beta-globin. Beta-globin makes up most of the hemoglobin in the human body. Hemoglobin is a protein required for the red blood cells to be able to carry oxygen throughout the body. These results are great because now patients will not need to continuously receive blood transfusions to be able to carry out normal red blood cell functions.
Blubird Bio had a 52-week low of $17.40 per share and now trades around $81 per share. The share price of Blubird Bio has been climbing, and after yesterday's results with treating beta-thalassemia major, rightfully so. The company is poised to do well in treating rare genetic disorders because of its gene-therapy technology. Bluebird Bio though also has a collaboration with Celgene (CELG) in which Bluebird Bio is using its CAR-T technology to treat hematologic malignancies and solid tumors. This CAR T cell therapy allows the company to modify the genes of T-cells and then place them into the patients body where they are able to target and destroy only cancerous cells in the patient's body. Bluebird Bio is set up well in gene therapy and we believe that additional catalysts may bring to additional pops in the share price over the coming months.
