December 9, 2014 / 4:10 PM / 5 years ago

Bluebird shares leap after gene therapy seen as cure for blood disorder

(Reuters) - Shares of drug developer bluebird bio Inc shot up on Tuesday, after the company presented data that showed a cure for beta-thalassemia is within reach, prompting several brokerages to raise their price targets on the stock.

The Cambridge, Massachusetts-based company’s stock jumped about 72 percent to $84.00 in early trade.

One of the most common genetic blood disorders, beta-thalassemia, is characterized by a reduction in hemoglobin, and requires patients to take lifelong transfusions. If left untreated, those affected often die in their forties.

Data presented at the American Society of Hematology meeting on Monday showed that four beta-thalassemia patients transplanted with bluebird’s LentiGlobin product, including one with the most severe form of the disease, were essentially cured.

The therapy resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support, bluebird said.

This data represents a “quantum leap” in the management of this disorder by shifting from “life-long symptom management to once-and-done cures,” Roth Capital Partner’s Debjit Chattopadhyay said, raising his price target on the stock to $90 from $50.

LentiGlobin aims to treat beta-thalassemia and sickle cell disease, another blood disorder, by inserting a gene into the patient’s own stem cells and then transplanting those modified cells back through infusion.

LentiGlobin’s results read-through favorably to the sickle-cell indication, Piper Jaffray analysts said, raising their target to $112 from $52.

“With 100,000 sickle patients in the United States and a likely gene therapy price point upwards of $500,000, this is a $50-100 billion U.S. opportunity alone.”

Suntrust Robinson increased its target to $115 from $57, while Wedbush Securities raised theirs to $94 from $51.

Meanwhile, luspatercept, being developed by Acceleron Pharma Inc and Celgene Corp, was found to treat both the anemia and complications of beta-thalassemia, such as iron overload.

This mid-stage data “represents a potentially soft treatment option for patients not willing to undergo the complex gene therapy protocol,” Chattopadhyay said.

Up to Monday’s close, bluebird had risen more than 133 pct this year.

Reporting by Natalie Grover in Bengaluru; Editing by Don Sebastian

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