Alexis Thompson, MD, MPH, on Beti-Cel's Approval in β-Thalassemia


The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed beti-cel's approval.

“This is an extraordinary day, to see the approval of the first gene therapy for transfusion dependent beta thalassemia. In my opinion, this is really a breakthrough and considerably transformational not only for thalassemia but also for the field.”

The FDA has approved betibeglogene autotemcel (beti-cel; bluebird bio), now marketed as ZYNTEGLO, a gene therapy for adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions (transfusion dependent thalassemia; TDT). The approval is based off positive data from multiple studies, including the ongoing phase 3 HGB-207 study (NCT02906202) in which 20 of 22 evaluable patients (91%), including 6 of 7 patients (86%) younger than 12 years of age, reached transfusion independence.

Beti-cel is an autologous gene-edited cell therapy that uses a lentiviral vector to deliver a modified form of the β-globin gene into patients’ hematopoietic stem cells ex vivo which are then infused back into the patient. The therapy enables normalized production of hemoglobin without regular RBC transfusions. The therapy will only be available at qualified treatment centers with expertise in stem cells, cell and gene therapy, and β-thalassemia.

CGTLive spoke with Alexis Thompson, MD, MPH, primary investigator and chief, division of hematology, Children’s Hospital of Philadelphia, to learn more about the approval and how it changes the treatment landscape for patients with TDT.

FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News release. FDA. August 17, 2022.
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