Precious Metals

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>>> Bluebird Bio: A shot at the big time



http://www.fool.com/investing/2016/06/29/these-3-biotechs-are-down-more-than-36-this-year-a.aspx?source=yahoo-2&utm_campaign=article&utm_medium=feed&utm_source=yahoo-2



While most therapies require lifelong treatment, Bluebird is on the cusp of providing a cure for a small set of patients with a big problem. Childhood cerebral adrenoleukodystrophy (CCALD) is deadly inherited disorder caused by a faulty gene that affects roughly 40 boys born in the U.S. each year.

A stem-cell transplant from a donor with a functional copy of the gene is a potential cure, but it's also frequently fatal because of graft-vs.-host disease. Bluebird's solution is to avoid graft-vs.-host complications by harvesting these patients' own cells, and using its lentiviral vector-based candidate, Lenti-D, to "infect" the cells with a functional copy of the faulty gene. Instead of receiving donor cells, patients receive their own, and the results so far are terrific. Lenti-D patients are performing on par with donor cell-receiving patients, but without any deadly side effects.

With such a small population of CCALD patients, an approval in this indication won't make Bluebird profitible. It would, however, provide regulators plenty of real-world safety data with its lentiviral-based cell therapies, which could help its Lentiglobin candidate earn approval for two much larger indications.

Hemoglobin gene disorders beta-thalassemia and sickle-cell disease affect about 288,000 and 25 million patients worldwide, respectively. They are somewhat treatable by means safer than donor stem-cell transplants, but the outcomes are generally poor. With potential safety data from Lenti-D, Lentiglobin has a chance of earning approval to cure enough patients to send Bluebird's bottom line soaring.

With the company's shot at the big time, its current enterprise value (a theoretical purchase price that considers a company's debt and cash) of just $916 million is a bargain.

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