The company broke news on Saturday, when it announced that Two patients with a serious, inherited blood disorder have been able to stop blood transfusions following a single treatment with an experimental gene therapy developed by BLUE. The patients reported have beta-thalassemia (B-thal), a disease caused by a missing or defective gene which prevents oxygen-carrying hemoglobin from functioning properly. The Bluebird therapy, also called LentiGlobin, replaces the defective gene with a functional one.
"Following the (gene) transplant, we're seeing near normal levels of hemoglobin. These early results far exceeded our expectations," said Chief Medical Officer David Davidson.
The company estimates, that there are about 15,000 patients with B-thal in the U.S. and Europe, and 270 000 in other parts of the world, with 60 000 new cases each year. In addition, they therapy has also many other uses, which might be the reason Celgene (CELG) is partnering with them (10-K):
"We believe that our vectors can be used to introduce virtually any gene into a cell and have the potential to be manufactured on a commercial scale reproducibly and reliably, as each new vector is produced using substantially the same process."
The stock shot up 32%, reaching a new all-time high and a market cap of $844 million. It hasn't been bought by insiders since it's IPO. The total float is 22 million while 5.7% of that is short. The company has currently 192 million in cash, which they believe will be enough until at least the end of 2015.