Bluebird Bio Inc (NASDAQ: BLUE) has presented this Saturday a highly anticipated data on a progress of the first patient with severe sickle cell disease (SCD) who is treated with the company’s main drug candidate, LentiGlobin, as well as an update on two patients with beta-thalassemia. The data was announced at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria. Today the stock of the company surged about 10% in pre market trading hours.
Sickle cell disease and thalassemia are inherited genetic disorders, caused by errors in the genes responsible for carrying oxygen within the red blood cell. Both disorders cause fatigue, jaundice, and episodes of pain that often reach very severe levels. Since the diseases are inherited, usually both parents must pass on an abnormal gene for a child to have one of the disorders. Both disorders are, at times, fatal.
Bluebird Bio is clinical-stage biotechnology company that develops several innovative gene therapies for severe genetic and orphan diseases. LentiGlobin is a sophisticated one-time gene therapy designed to target and replace abnormal genes.
In order to survive sickle cell and beta-thalassemia, patients must undergo regular monthly transfusions of blood to replace damaged red blood cells with new ones. The new data revealed that the French teenager suffering from severe sickle cell who was initially treated with LentiGlobin continues to improve without any transfusions and didn’t show symptoms of the disease returning.
Data from the two beta-thalassemia patients also showed impressive results. The two patients with beta-thalassemia didn’t rely on transfusion for 14 and 16 month. The data shows that as of May 2015 both patients have near normal hemoglobin levels. David Davidson, M.D., chief medical officer, of bluebird bio comments, “We are excited that the promising early results for LentiGlobin in beta-thalassemia major are now extending to the first treated patient with severe sickle cell disease. With the treatment of the first patient with severe sickle cell disease in the HGB-206 study, we look forward to gaining increasing clarity on the potential clinical benefit of LentiGlobin for patients with severe sickle cell disease.”