Bluebird Bio Inc (NASDAQ:BLUE) moved skyward on stock markets Monday, on new product developments.
The company presented new results for betibeglogene autotemcel (beti-cel), investigational gene therapy for β-thalassemia (beta-thal) who require regular red blood cell (RBC) transfusions.
New data demonstrate that adult and pediatric β-thalassemia patients can produce normal or near-normal total hemoglobin levels, remain transfusion-free, and achieve stable iron markers through up to seven years of follow-up (n=3).
"In the analysis published in New England Journal of Medicine, 91% of patients, including six of seven patients under the age of 12, achieved transfusion independence as well as improvement in erythropoiesis and liver iron concentrations," said the survey’s lead author, Professor Franco Locatelli, lead author.
Beti-cel stabilized iron markers in patients who achieved transfusion independence and could stop iron chelation (n=20/34), with markers of iron management improving toward normal.
Adult, adolescent, and pediatric patients experienced early and sustained improvement in health-related quality-of-life measures from baseline across Phase 3 studies.
Sickle cell disease is a serious, progressive and debilitating genetic disease caused by a single mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin.
In the U.S., the median age of death for someone with sickle cell disease is 43-46 years. Additionally, one in four people living with sickle cell disease experiences a stroke by age 45.
BLUE shares are up 49 cents, or 5.5% at $9.45 on Monday.
