Bluebird Bio's In The Pink...

(RTTNews) - It's been an absolute banner year so far for gene therapy developer bluebird bio Inc. (BLUE) whose share price have surged more than 130 percent year-to-date.

Before we discuss what's making news at bluebird bio today, here's what "gene therapy" means in simple terms.

Gene therapy is a technique that uses genes to treat or prevent diseases caused by defective or missing genes. This approach helps to address the underlying cause of the disease, rather than offering solutions that focus only on the disease symptoms. Though not a new field, gene therapy is still considered experimental as it faces many technical challenges and concerns regarding its safety.

Did you know the world's first commercial gene therapy was approved in China?

Gendicine, developed by SiBiono GeneTech Co., Ltd., for head and neck squamous cell carcinoma was launched in China in 2004. A year later, another gene therapy product - Oncorine for the treatment of head and neck cancer - was launched, in China, by Shanghai Sunway Biotech Co. Ltd.

Russia launched its first gene therapy drug, Neovasculgen, to treat Peripheral Arterial Disease on the market in 2011.

In the Western world too, there is one approved gene therapy - Glybera - developed by uniQure N.V. (QURE) for adult patients diagnosed with familial lipoprotein lipase deficiency. Approved by the European Commission in November of 2012, Glybera is expected to be launched in Europe this quarter or in Q1, 2015. It is currently not approved for use outside of the European Union.

Now, coming back to today's topic under consideration - bluebird bio - its stock was up more than 43% in Monday's extended trading (Dec.8, 2014), following promising results of its investigational gene therapy LentiGlobin BB305 in patients with beta-thalassemia major, a severe form of beta-thalassemia. The results were presented at the Annual Meeting of the American Society of Hematology yesterday.

Beta thalassemia is an inherited blood disease that reduces the production of hemoglobin. Abnormalities in a gene that is responsible for the production of beta globin (beta-T87Q-globin) causes sickle-cell disease and beta thalassemia. Depending on the severity of symptoms, beta thalassemia is classified as thalassemia major (also known as Cooley's anemia) and thalassemia intermedia.

Beta globin is one of the proteins that make up hemoglobin. Treating beta-thalassemia includes frequent and lifelong blood transfusions, which deliver red blood cells to the body to correct the anemia.

bluebird bio's gene therapy LentiGlobin BB305 involves inserting a fully functional human beta-globin gene into the patient's own hematopoietic stem cells ex vivo and then transplanting those modified cells into the patient through infusion, also known as autologous stem cell transplantation.

LentiGlobin BB305 is under two phase 1/2 trials namely, Northstar Study and HGB-205 Study.

The Northstar Study is evaluating the safety and efficacy of LentiGlobin BB305 for the treatment of subjects with beta-thalassemia major and the HGB-205 Study is evaluating the safety and efficacy of LentiGlobin BB305 in the treatment of subjects with beta-thalassemia major and severe sickle cell disease.

The Northstar Study, designed to enroll up to fifteen subjects, was initiated in March of this year. As of December 1, 2014, five subjects with beta-thalassemia major have undergone infusion with LentiGlobin BB305.

The HGB-205 study, designed to enroll up to seven subjects, was initiated in France last December. As of December 1, 2014, two subjects with beta-thalassemia major have undergone infusion with LentiGlobin BB305 drug product.

As recently as October of this year, the first patient with severe sickle cell disease was infused with LentiGlobin BB305 drug product in a separate phase I study (HGB-206) in the United States. This study is designed to enroll up to eight subjects.

Based on data from bluebird bio's ongoing Northstar and HGB-205 trials, the first four patients with beta-thalassemia major treated with LentiGlobin BB305 drug product, who had at least three months of follow up, are transfusion-free, which means the patients are producing robust levels of beta-T87Q-globin.

The company expects to complete patient enrollment for the Northstar and HGB-205 studies in 2015.

bluebird bio will host a conference call and webcast on December 10, 2014 to review the data presented at the American Society of Hematology.

Stay tuned...

BLUE closed Monday's trading 9.35% higher at $48.89. In after-hours, the stock was up 43.18% at $70.