What if you could
let go of your transfusions?

ZYNTEGLO® has the potential to free people with beta-thalassemia from regular transfusions.

Explore the Clinical Results
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Beta-thalassemia transfusion independence Beta-thalassemia transfusion independence

Lifelong transfusions treat the symptoms of beta-thalassemia but do not address the underlying genetic cause

ZYNTEGLO is a gene therapy that uses gene addition to help your body produce functional adult hemoglobin, potentially eliminating the need for regular transfusions.

Specifically made for each person, ZYNTEGLO uses a person’s own blood stem cells and adds working copies of the beta-globin gene (needed to create functional adult hemoglobin) to the person’s cells. This may allow the person to produce sufficient hemoglobin.

Who is ZYNTEGLO for?

ZYNTEGLO is a one-time gene therapy to treat beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions

Pediatrics and Adults
No Donor Required
All Genotypes (β0/β0 and Non-β0/β0)
  • β00: no beta-globin is produced
  • Non-β00: a reduced amount of beta-globin is produced

*In the phase 3 studies, the safety and efficacy of ZYNTEGLO was studied in patients aged 4–34 years.
Made using the patient’s own stem cells.

In two phase 3 studies, about 9 out of 10 (89%) patients treated with ZYNTEGLO stopped transfusions

Transfusion independence (TI) meant that patients achieved a weighted average hemoglobin of ≥9 g/dL without any transfusions for a continuous period of ≥12 months at any time during the study after infusion of ZYNTEGLO.

The majority of patients achieved transfusion independence (89%; 32/36 patients) and had a normal or near-normal median total hemoglobin of 11.5 g/dL.

Normal Hb ranges for healthy individuals from the American College of Clinical Pharmacy:

  • 2–9 years: 11.5–14.5 g/dL
  • Males 10–17 years: 12.5–16.1 g/dL
  • Females 10–17 years: 12–15 g/dL
  • Male adults (≥18 years): 13.5–18 g/dL
  • Female adults (≥18 years): 12.5–16 g/dL

The phase 3 studies evaluated the efficacy of ZYNTEGLO in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for transfusion independence.

More on Clinical Studies

The Treatment Journey

Getting treatment with ZYNTEGLO is a multi-step process. Find information you need and get answers to your questions before getting started.

Get support and helpful resources with my bluebird support

If you’re considering ZYNTEGLO, we want to make sure you’re supported. That’s why we’ve created my bluebird support—a collection of educational resources aimed at helping you or someone you care for stay informed for any decision-making process with your doctor.


If you have a child or loved one with beta-thalassemia who requires regular transfusions, have you talked to them about their goals for the future? By encouraging an open dialogue with questions, you can learn more about how they feel their disease plays a role in their plans.
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