Bluebird Bio Presents Promising Results for Gene Therapy in Rare Blood Disorder

Bluebird Bio Presents Promising Results for Gene Therapy in Rare Blood Disorder

Source: 
CP Wire
snippet: 
  • One time treatment may address underlying cause of the disease
  • Works by inserting a functional human beta-globin gene into a patient’s own hematopoietic stem cells
  • Evaluating LentiGlobin in both β-thalassemia and Sickle Cell Disease
  • LentiGlobin granted orphan drug status in both β-thalassemia and Sickle Cell Disease