As reported in Xconomy, Bluebird Bio reports on a successful, single application of its experimental gene therapy treatment for beta-thalasemmia in this month's Nature. Earlier this year, both Genzyme and Third Rock Ventures invested $35 M in the company based on its gene therapy promise.
The patient in the recent Nature study had been forced to have monthly blood transfusions to treat his disease since he was 4 years old. Post-gene therapy, he is now able to produce enough of his own hemoglobin to forgo any blood transfusions. In fact, he's now holding down a full time job as a cook in Paris.
Bluebird is now encouraged enough to recruit more patients for a larger study. Their treatment relies on the use of a lentivirus to deliver a copy of a gene to make hemoglobin properly. Bluebird uses the patient's own adult stem cells and injects them in vitro with the virus. It then reinfuses the patient with their own reprogrammed cells. In this case, it worked. Now, Bluebird needs to show it can do it on a larger population without any severe side effects.
If they can, then Genzyme, Third Rock and thalassemia suffers will all be happy.
Posted by Bruce Lehr September 15th 2010.