What is Zynteglo?

ZYNTEGLO^® is an FDA-approved gene therapy from Bluebird Bio that has the potential to free people with beta-thalassemia from regular transfusions. This gene therapy is a one-time treatment that addresses beta thalassemia at the genetic level and is approved for adult and pediatric patients 12 years old or older who require regular blood cell transfusions. This treatment is crafted from the patient’s own modified cells to generate a functional beta-globin.

Children’s Health is one of a few designated Qualified Treatment Centers (QTCs) centers in the country able to provide Zynteglo.

How does Zynteglo work?

Blood stem cells are collected from your body and sent to a lab to begin manufacturing. Zynteglo is manufactured by adding working copies of the beta-globin gene to your collected cells. Your modified stem cells (Zynteglo) are then returned to your body through an IV infusion.

For more detailed information about Zynteglo please visit the manufacturer’s website.

How can a child receive this treatment?

Children’s Health is not able to administer Zynteglo to patients unless their health insurer has established coverage guidelines. We also cannot begin the process for authorization until your insurance carrier has issued guidelines. For a step-by-step guide for navigating this authorization process, please call our CCBD Clinic at 214-456-2382.

As of today, the following insurers have established coverage guidelines for Zynteglo. We are awaiting guidelines from all other insurance carriers, and we are hopeful additional guidelines will be released soon.

If your insurer has not issued guidelines, here are two things you can do:

1. We encourage you to contact your insurance carrier and advocate for your child to receive Zynteglo
2. If your insurance plan is through an employer, we also suggest you contact your employer’s HR personnel and ask them to also advocate for your child with contacts they may have at your insurance provider.
   • If you are a Texas Medicaid recipient, we recommend that you call and advocate for coverage.

We know this can be a challenging waiting process as insurance carrier guidelines are created. Children’s Health will continue to stay updated on new information so that we can support our patient families in their steps toward receiving Zynteglo.

If you have additional questions, please contact the CCBD Clinic at 214-456-2382.