New Data At ASH21 Show Bluebird's Thalassemia Gene Therapy Potentially Curative One-Time Treatment

Bluebird Bio Inc BLUE 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 (NEJM), 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 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.
• Related Link: Bluebird Bio's Thalassemia Gene Therapy Under Priority Review With FDA.
• Price Action: BLUE shares are up 9.93% at $9.85 during the premarket session on the last check Monday.
• Check out our coverage of the American Society of Hematology (ASH) Annual Meeting 2021 here.