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ZYNTEGLO® Treatment

Children’s National Hospital offers ZYNTEGLO® (betibeglogene autotemcel), an FDA-approved gene therapy for transfusion-dependent beta thalassemia, which is an inherited blood disorder that causes the body to make less hemoglobin, resulting in anemia.

This unique therapy is made specifically for each child or adult, by adding functional copies of the beta-globin gene to their own blood stem cells. Most patients with beta thalassemia who have received a one-time ZYNTEGLO® treatment have been able to produce sufficient hemoglobin because of the treatment, freeing them from regular blood transfusions.

The ZYNTEGLO® Treatment Process

At Children's National, our dedicated team of Blood and Marrow Transplantation experts can discuss your child's treatment history and overall health to see if they are a candidate for ZYNTEGLO® therapy.

The treatment process will include the following stages over several months:

  • Collection. Blood stem cells are collected from your child’s body and sent to a lab to begin manufacturing.
  • Modification. At the lab, ZYNTEGLO® is manufactured by adding working copies of the beta-globin gene to your child’s collected cells.
  • Chemotherapy. Prior to the ZYNTEGLO® infusion, your child will undergo several days of chemotherapy in the hospital to make room for new stem cells to grow.
  • Infusion. Your child’s modified stem cells (ZYNTEGLO®) are returned to their body via IV infusion. The cells move through their bloodstream to your child’s bone marrow and produce new stem cells in a process called engraftment.
  • Production. The new cells contain working copies of the beta-globin gene, leading to the production of functional hemoglobin.
  • Monitoring and recovery. After your child’s infusion, they will stay in the hospital for 3-6 weeks to manage side effects of chemotherapy and monitor for infection until engraftment occurs.  

Contact Information

If you have questions or would like more information about ZYNTEGLO® at Children’s National, please call 202-476-4267 (select option 3) or email us.

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Treatment for International Patients

Children’s National Hospital is one of only a few qualified treatment centers in the United States that offer the ZYNTEGLO® therapy to patients with beta thalassemia. Families from outside of the United States may email our Global Services team to inquire about enrolling your child in our ZYNTEGLO® treatment program.

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