Typically seeing an experimental treatment work against a single patient doesn't count for much in the drug development world. But when a gene therapy appears to cure a genetic blood disorder for more than two years, heads turn.
That was the case for Cambridge, MA-based Bluebird Bio, formerly known as Genetix, which achieved a major milestone when the Journal Nature published a study outlining the case of a 21-year-old Parisian suffering from beta thalassemia--which reduces red blood cell counts--who was able to end the monthly blood transfusions he had needed from the age of three. Now Bluebird plans to treat nine more patients with thalassemia or sickle cell anemia to see if it can replicate the results. And it's basking in the limelight after grabbing some prominent headlines for the success.
"Major journals don't publish papers very often based on one patient but this is a significant milestone," bluebird CEO Nick Leschly tells Bloomberg. "Thalassemia doesn't just go away, so if you get a remission it means the treatment has worked."
"This is a very significant study. Beta thalassemia is really challenging for gene therapy,'' Guangping Gao, the director of the gene therapy center at the University of Massachusetts Medical School, tells the Boston Globe. "One patient is a very small study, but this is really a milestone.''
Bluebird won $35 million in backing from Third Rock Ventures and Genzyme back in the spring--right in the venture group's sweet spot. Third Rock likes to provide substantial early rounds to biotech companies which often fall in a range of $30 million to $40 million.