Cincinnati Children’s recently infused its first two patients with Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

The medical center is one of only 10 in the United States to earn designation as a Qualified Treatment Center for Zynteglo. The U.S. Food and Drug Administration approved Zynteglo in August 2022 after a fast-track review. Researchers established the treatment’s effectiveness in two multicenter clinical studies. Of the 41 patients who received Zynteglo in those studies, 89% achieved transfusion independence. The remaining patients had a partial response.

“This gene therapy offers curative potential for patients with transfusion-dependent beta thalassemia who don’t have a suitable donor to undergo a hematopoietic stem cell transplant [HSCT],” says Michael Grimley, MD, medical director for the Division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children’s.

A Potential Alternative to Lifelong RBC Transfusions, Health Risks

Beta thalassemia is very rare and typically diagnosed in the first months of life after patients present with anemia. Children and adults with the most severe form of this disease need monthly RBC transfusions and must take daily medication to prevent iron overload. Even with exceptional medical care and compliance, people with beta thalassemia face the risk of severe organ damage and a significantly shortened lifespan due to the need for chronic RBC transfusions and subsequent iron overload.

“Allogeneic HSCT is curative, but a significant number of patients with transfusion-dependent beta thalassemia lack an HLA-matched donor,” Grimley says. “This new gene therapy is an exciting breakthrough for people who otherwise face a lifetime of monthly blood transfusions and associated health risks.”

Zynteglo is available to patients ages 4 to 34 who qualify. To learn more, please contact Melissa.Hunter@cchmc.org or call 513-803-1962.