It’s been two years since disappointing clinical trial results raised questions of whether Bluebird Bio (BLUE) was capable of developing a one-shot gene therapy cure for sickle cell disease. On Wednesday, the biotech disclosed the first preliminary data suggesting a significant obstacle may have been cleared.
The new data show that Bluebird has been able to increase the number of gene-corrected cells taking hold in the bone marrow of sickle cell patients by improving the manufacturing process for its gene therapy, known as LentiGlobin. That suggests the patients may be able to produce more normal red blood cells, rather than damaged, sickled cells.
But so far, Bluebird has only collected data from two sickle cell patients treated with its improved LentiGlobin, and neither of them has been followed long enough to determine if there are enough normal red blood cells to cure the disease.
About the Author Reprints
Today, Celgene and Bluebird announced that bb2121 has been granted accelerated review status in the U.S. for the treatment of multiple myeloma. http://alph.st/bfbe4ee3