Positive β-thalassemia results provide rare cheer for bluebird

Long-suffering investors in bluebird bio (Nasdaq: BLUE) had a rare reason for cheer as the company presented positive results for betibeglogene autotemcel (beti-cel) at the American Society of Hematology (ASH) Annual Meeting.

The new data on the deeply studied investigational gene therapy demonstrate adult and pediatric patients living with β-thalassemia (beta-thal) who require regular red blood cell (RBC) transfusions can produce normal or near-normal levels of total hemoglobin and continue to remain transfusion-free, and achieve stable iron markers, through up to seven years of follow-up.

These findings further support beti-cel as a potentially curative one-time treatment option that addresses the underlying genetic cause of beta-thal and mitigates the burdens associated with the practical management of the disease.