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New data published in NEJM further demonstrates beti-cel as a potentially curative one-time gene therapy for beta-thalassemia patients.

Read time: 1 mins
Published:13th Dec 2021
bluebird bio, Inc. presented new results for betibeglogene autotemcel (beti-cel), a deeply studied investigational gene therapy, that demonstrate adult and pediatric patients living with beta-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 (n=3).

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. The data were highlighted in the press program and will be delivered in an oral presentation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 11-14, 2021, at the Georgia World Congress Center in Atlanta and virtually. Data from the pivotal HGB-207 Northstar-2 study were also simultaneously published in an original article in The New England Journal of Medicine (NEJM) titled, “Betibeglogene Autotemcel Gene Therapy for Non-beta/beta0 Genotype betaThalassemia.”

See-"Betibeglogene Autotemcel Gene Therapy for Non–beta0/beta0 Genotype beta-Thalassemia"-Franco Locatelli, M.D., Ph.D., Alexis A. Thompson, M.D., M.P.H., Janet L. Kwiatkowski, M.D., John B. Porter, M.D., et al. December 11, 2021. DOI: 10.1056/NEJMoa2113206.

Condition: Beta-thalassemia
Type: drug

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