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Biotech Developing Genetics Therapies Gains $101M in IPO

DNA strand (NSF)

(James. J. Caras, National Science Foundation)

The biotechnology company bluebird bio in Cambridge, Massachusetts developing therapies for genetic disorders raised $101 million through its initial public offering (IPO) of 5.9 million shares priced at $17.00 a share. The company’s stock, trading on the NASDAQ under the code BLUE was priced at $26.00 a share at 11:00 am ET today.

bluebird bio develops treatments for severe genetic and rare diseases with a technology based in part on the work of co-founder Philippe Leboulch, a researcher and lecturer at Paris University School of Medicine, Harvard Medical School, MIT, and Brigham and Women’s Hospital in Boston. Leboulch serves as a scientific advisor to bluebird bio. The company’s technology takes a patient’s own hematopoietic (bone marrow) stem cells and cultures them outside the body to create healthy replacement genes for the mutated stem cells causing the disease.

The lead product for bluebird bio is Lenti-D, a one-time treatment to stabilize and stop the progression of childhood cerebral adrenoleukodystrophy, a rare inherited disorder that results in the breakdown of the myelin sheath protecting nerve cells in the brain. The disease affects mainly males and causes the loss of motor coordination, visual and hearing disturbances, reduced cognitive function  dementia, seizures, and adrenal dysfunction. The only current treatment for the disease — which was the subject in the 1992 feature film Lorenzo’s Oil — is a transplant of healthy hematopoietic stem cells from siblings or non-sibling donors, but non-siblings with acceptable stem cells are available for less than 30 percent of patients.

Lenti-D received an orphan drug designation from the FDA and European Medicines Agency. The therapy has undergone early stage (phase 1 and 2) clinical tests and the company is preparing intermediate and later stage clinical trials for later this year.

Another product under development is LentiGlobin to treat beta-thalassemia and sickle cell disease. Beta-thalassemia is an inherited blood disorder that results from an abnormal beta-globin gene causing reduced beta chains of hemoglobin leading to defective red blood cells. Sickle cell disease is also an inherited blood disease caused by a mutated beta-globin gene, resulting in abnormal red blood cell function and chronic anemia.

LentiGlobin therapy inserts functioning human beta-globin genes into the patient’s own hematopoietic stem cells. Early proof-of-concept tests in 2010 on a single patient in France showed positive results. bluebird bio plans to expand that study and start an early-stage clinical trial in the U.S. in 2013.

The company is collaborating with Celgene Corporation to develop gene therapies to treat cancer, taking a patient’s blood and extracting immune-system T-cells. Those T-cells are then genetically modified and reintroduced into the patient to recognize and attack cancer cells. bluebird will be responsible for development of the technology through early-stage clincial trials, with Celgene having the option to license products from the collaboration. Celgene and bluebird bio also plan to collaborate with Baylor College of Medicine to evaluate the technology with blood-related and solid tumor cancers.

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Hat tip: Fortune/Term Sheet

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