Understanding gene therapy.

There are several types of bleeding disorders, including hemophilia A (factor VIII deficiency) and hemophilia B (factor IX deficiency). People with these bleeding disorders have a mutated (changed) gene that affects how the body makes a clotting factor. A clotting factor is a protein that helps blood to clot. With hemophilia A and hemophilia B, the gene that tells the right cells to make factor VIII or factor IX has changed. With the mutated gene, you have a missing factor or one that doesn’t work well. This makes it harder for blood to clot and bleeding to stop.

Treatment options.

Several medications are available to treat bleeding disorders, and more are on the way. Each drug works in its own way to help prevent or treat bleeding episodes. But none of them target the actual cause of a bleeding disorder, which is the mutated gene. That’s where gene therapy can help.

As of now, two gene therapy options are approved by the Food and Drug Administration (FDA) for a bleeding disorder. Hemgenix^® is approved to help treat hemophilia B in adults. Roctavian™ is approved to treat severe hemophilia A in adults. There’s still a lot of research to be done for gene therapy. But, it’s a promising treatment option for bleeding disorders. Here’s some helpful information about gene therapy. 

The role of gene therapy.

Genes tell cells to do certain things. Sometimes genes mutate, which causes them to not work well. In bleeding disorders, mutated factor genes make it hard for blood to clot. Gene therapy may help treat bleeding disorders by replacing the mutated gene with a healthy one. Right now, someone with a bleeding disorder can receive gene therapy only one time.

How it works.

It’s not easy to get healthy genes into cells. Healthy genes can only get into cells with the help of a “delivery vehicle”. This delivery vehicle is called a vector. Today, most vectors are viruses. The viruses are altered so they don’t carry the parts that cause illness. That means you won’t get sick from a viral vector. Viruses are used as vectors because they can easily get into cells. The vector carries the working gene into the cell, delivering the package of genetic instructions. This allows the cells to work properly, so the missing factor can be made by the body. .

What happens after.

After gene therapy, you’ll have close follow up for many years. Your health care team will check for safety and track how well the gene therapy is working. They’ll also collect information that may help other people with a bleeding disorder who are thinking about gene therapy.

Talk to your doctor or CVS Specialty^® CareTeam to learn more about gene therapy.