Bluebird Bio’s In The Pink…

It's been an absolute banner year so far for gene therapy developer bluebird bio Inc. (BLUE: Quote) whose share price has 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.

Go here to see the original:
Bluebird Bio's In The Pink...