Gene therapy edits your child’s genetic code to modify their disease. Our genetic code is the set of instructions present in every cell of our body. They tell the cells how to act and are the instructions for our body’s various parts.

In a monogenic disease, a change in a single gene causes certain cells to go awry. Cells and genes are complex, so there are lots of ways things can go wrong.

Gene therapies aim to fix these diseases by changing the genetic code. They may edit the gene, add an additional gene or make some other genetic change to the body’s cells to fix the issue caused by the gene change.

We currently offer:

• Casgevy, which is for people 12 years and older with sickle cell disease or beta thalassemia

Other gene therapies may be available as clinical trials. Ask your child’s care team if there are any clinical gene therapies they qualify for.

Gene therapies are some of the most expensive and complex treatments. Cost and insurance coverage can feel like big hurdles, and that’s normal. But you won’t face it alone.

Our financial counselors and care team will work closely with your family and insurance provider. We can help you understand your options and get the support you need. Many families have been able to access treatment with the right help.

Casgevy is a gene therapy for severe sickle cell disease and transfusion-dependent thalassemia. It can be life-changing. The Food and Drug Administration approved it in 2023. MACC Fund Center for Cancer and Blood Disorders at Children’s Wisconsin is one of the few dozen sites in the United States that can offer this therapy.

It works by making a gene change that increases how much fetal hemoglobin the body makes. Fetal hemoglobin makes up for the broken adult hemoglobin that causes sickle cell and thalassemia. Most kids see their symptoms improve drastically.

We make Casgevy from your child’s own blood stem cells. The company makes the gene change and grow up the changed cells. They’re then returned to your child in a single-dose infusion as part of a stem cell transplant.

The FDA approved Casgevy for people 12 years and older with:

• Sickle cell disease with repeated pain crises
• Transfusion-dependent beta thalassemia

It’s for kids and young adults whose symptoms are severe and don’t go away with standard treatments.

Choosing gene therapy is a big decision.

Ask yourself:

• Has my child’s condition been hard to control with current treatments?
• Are we ready for an intense process that includes chemotherapy and a hospital stay?
• Do we understand the risks, side effects and long-term follow-up required?
• Does my child have a strong support system for recovery?
• Are we looking for a possible long-term solution — or even a cure?
• Can we manage the cost and logistics of treatment?

Comprehensive care: The MACC Fund Center for Cancer and Blood Disorders is here for you and your family through every step of your gene therapy journey. Our team includes experts in diagnosing and treating sickle cell and other blood disorders.

Specialists in sickle cell and thalassemia: With more than 400 patients, we are the state's largest provider of sickle cell care. We also have the state's largest pediatric thalassemia program. Our team of experts provides the best care for kids living with these blood disorders. We offer tailored care plans, help manage side effects and long-term care.

Clinical trials: Our team offers dozens of clinical trials. These drive the next generation in treatment options, including new gene therapies. Our patients get access to the latest treatments. Our clinical trial work helps children with blood disorders worldwide.

Expert partners: We are in the Central Region Thalassemia Consortium. We are also a Cooley's Anemia Foundation provider. These partnerships give our kids access to the latest care standards and national support. We provide your child with the best possible care, including access to new therapies, research and clinical trials.