Alexis Thompson, MD, MPH, on the Impact of Beti-Cel's Approval in Beta Thalassemia


The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed the collaboration and research that went into developing beti-cel.

“This has really been a great day for science. This has been a collaboration that's been international, that has involved hematologists, general pediatricians,internists, researchers in the gene therapy field, and a remarkable commercial sponsor. My deepest gratitude is to the patients from the phase 1 trial all the way through the pivotal phase 3 trial that had the courage to be on this journey with us and to be able to pave the way for other patients with transfusion dependent beta thalassemia.”

The gene therapy ZYNTEGLO (betibeglogeneautotemcel; beti-cel; bluebird bio) has been approved by the FDA foradult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions (transfusion dependent thalassemia; TDT).1 Qualifying treatment centers (QTCs), experts with stem cells, TDT, and gene therapies, will be able to administer the therapy.

ZYNTEGLO delivers a modified form of the β-globin gene into patients’ hematopoietic stem cells and then back into the patient. It is an autologous gene-edited, lentiviral cell therapy that enables normalized production of hemoglobin without regular RBC transfusions.The therapy has demonstrated positive datain 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.

Most recently, the FDA Cellular, Tissue, and Gene Therapies Advisory Committee (AdComm) cast a unanimous vote in favor of the benefit of beti-cel in patients with TDT with a non–β0/β0 genotype, calling the efficacy of the drug "remarkable" and "life-changing".

CGTLive spoke with Alexis Thompson, MD, MPH, primary investigator and chief, division of hematology, Children’s Hospital of Philadelphia, to learn more about ZYNTEGLO and its benefit-risk profile. She discussed the previous AdComm meeting vote up to the approval and lauded the importance of the approval.

1. 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.
2. Cellular, Tissue, and Gene Therapies Advisory Committee June 9-10, 2022. Meeting. FDA. June 10, 2022.
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