bluebird bio Skyrockets Almost 70% On Blood Disease Data

December 9, 2014
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

A new gene therapy from biotech company bluebird bio was able to keep four patients with a lethal blood disorder from having to take life-sustaining blood transfusions within 90 days after they were treated, the company said this week.

Shares of Bluebird shot up almost 70 percent on the news in midday trading Tuesday, as the market looked eagerly for signs that Bluebird’s LentiGlobin BB305 could be a panacea for blood diseases.

Cambridge, Mass.-based Bluebird presented the data for the study at the American Society of Hematology conference in San Francisco Monday. It also reported that one patient had been able to forgo transfusions for a full 12 months at this point.

Bluebird’s LentiGlobin BB305 extracts blood stem cells and then infuses them with a working version of the malfunctioning gene that had caused the disease.

It treated four patients who were born with beta-thalassemia, a disease caused by a single gene that reduces or prevents the ability of red blood cells to produce hemoglobin, which is necessary to deliver oxygen to the body. People with the disease must undergo monthly blood transfusions in order to survive—but if Bluebird’s therapy continues to be successful, they may now be freed from that burden.

The number of people affected is not huge, but is certainly significant: Around 40,000 babies world-wide and between 1,000 and 3,000 in the U.S. are born with the condition each year.

The new therapy is so effective, Wall Street is champing at the bit to see it be rushed into later-stage trials to bring it to market more quickly.

“Bluebird plans to talk to U.S. and European Union regulators about speeding up the drug’s development by starting a later-stage study instead of waiting for 24-month data,” Chief Operating Officer Jeffrey Walsh told Bloomberg Tuesday.