Person living with beta thalassemia walking outside
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How CASGEVY® Works

People living with TDT pictured may or may not have received CASGEVY.

Noor, living with TDT

Noor, living with TDT

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People living with TDT pictured may or may not have received CASGEVY.

Before learning about how CASGEVY works, let's learn about the role that fetal hemoglobin plays in beta thalassemia.

Icon representing fetal hemoglobin

FETAL
HEMOGLOBIN

FETAL HEMOGLOBIN

Fetal hemoglobin is a type of hemoglobin that carries oxygen while you’re growing in the womb.

Icon representing the BCL11A gene telling the body to make less fetal hemoglobin and more adult hemoglobin

SHIFT TO ADULT
HEMOGLOBIN

SHIFT TO ADULT HEMOGLOBIN

Shortly after people are born, fetal hemoglobin starts to be replaced by adult hemoglobin. BCL11A is a gene that tells the body to make less fetal and more adult hemoglobin.

Icon representing fewer, smaller red blood cells

BETA THALASSEMIA AND HEMOGLOBIN

BETA THALASSEMIA AND HEMOGLOBIN

For people with beta thalassemia, their bodies are not able to make enough working adult hemoglobin. They have fewer, smaller red blood cells (RBCs) that are not as good at carrying oxygen.

Icon representing beta thalassemia symptoms starting

SYMPTOMS START

SYMPTOMS START

When the body makes less fetal hemoglobin, beta thalassemia symptoms tend to start.

CASGEVY is a gene therapy that is made by editing the BCL11A gene. CASGEVY is made with your body's own blood stem cells that are collected and sent for editing. So no donor is needed.

Editing outside of the targeted gene was not observed in the modified blood stem cells of people living with transfusion-dependent beta thalassemia (TDT) and healthy volunteers. The risk of editing outside of the targeted gene cannot be ruled out, due to differences in people's DNA. The impact of editing outside of the targeted gene is unknown.

Watch the video below to see how CASGEVY works in the body.

Watch the video below to see how CASGEVY works in the body.

CASGEVY® mechanism of action video
Information sheet with bar graph and line graph representing the study design and results
See the clinical study design and results

Learn how CASGEVY was studied.

Brochure icon for information about CASGEVY® (exagamglogene autotemcel)
Want to learn more about CASGEVY?

Check out the brochure for information about how CASGEVY may help.