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Wall Street Journal Highlights Promising Gene Therapy Advances

December 9, 2014
Yesterday, bluebird bio reported some very uplifting news at the annual meeting of the American Society of Hematology. Two patients with beta-thalassemia, a genetic disorder which normally requires regular blood transfusions, have been able to forgo transfusions for at least five months following a gene therapy treatment from bluebird.

The Wall Street Journal’s Ron Winslow reports:
Bluebird bio’s treatment involves extracting blood stem cells from a patient, treating them with a functioning version of the defective gene that is delivered in a viral delivery vehicle called a vector, then infusing the treated cells back into the body.

For the two patients, the treatment resulted in robust production of hemoglobin, researchers said.

“The early results are extraordinary,” said Alexis A. Thompson, head of hematology at Northwestern University’s Feinberg School of Medicine in Chicago, and lead investigator of the study. “In a relatively short period of time, they’re requiring minimal to no transfusion support.”

The first patient treated with a previous version of the treatment has now been transfusion-free for over six years, according to bluebird bio President and CEO Nick Leschly, who is also a member of BIO’s Board of Directors.

Advances such as this show the promise of biotechnology to transform lives and enable patients who had previously relied on frequent, onerous treatment regimens to enjoy newfound freedom and better health.  That’s the kind of progress that millions of men and women at biotechnology companies across the world are working hard to advance every day.

Read the full Wall Street Journal article here.