Beti-Cel Gene Therapy Leads to Transfusion Independence for Patients With TDT, Long-Term Data Confirms

Betibeglogene autotemcel (beti-cel), a Food and Drug Administration-approved gene therapy, helped 89% of patients with severe transfusion-dependent beta-thalassemia (TDT) achieve transfusion independence, according to findings from a long-term follow-up study published in The Lancet. Notably, there were no serious adverse events or deaths related to the gene therapy. 

Beti-cel, marketed as Zynteglo by Bluebird Bio, works by adding functional copies of a modified beta-globin gene (βA-T87Q-globin) into a patient’s own hematopoietic stem cells and progenitor cells (HSPCs), allowing them to produce normal to near-normal levels of hemoglobin without regular transfusions.

In this multicenter trial, 18 eligible patients with specific genotypes received beti-cel following busulfan-based myeloablative conditioning. Patients were observed for up to 24 months to monitor outcomes. As of January 2023, all study participants had enrolled in an ongoing 15-year follow-up to assess the durability of transfusion independence and the long-term safety profile of the therapy.

Representatives from Bluebird Bio plan to present the data at the upcoming 66th American Society of Hematology Annual Meeting and Exposition.