What if you could let go of your transfusions?

Beta-thalassemia transfusion independence

ZYNTEGLO is an FDA-approved gene therapy that has the potential to free people with beta-thalassemia from regular transfusions.

What if you could let go of
your transfusions?

ZYNTEGLO is an FDA-approved gene therapy that has the potential to free people with beta-thalassemia from regular transfusions.

Results from Clinical Studies

In two phase 3 studies, about 9 out of 10 (89%; 32/36 patients) treated with ZYNTEGLO achieved transfusion independence and had a normal or near-normal median total hemoglobin of 11.5 g/dL.*

Actor portrayals
Beta-thalassemia transfusion independence

Who is ZYNTEGLO for?

ZYNTEGLO is for adult and pediatric patients with beta-thalassemia who require regular blood transfusions.

The treatment journey

ZYNTEGLO is a one-time therapy with a highly coordinated process. Learn more about the treatment journey from start to finish.

Looking for support?

Download a brochure, view FAQs, or learn how my bluebird support Patient Navigators can help guide you through your treatment journey.
*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.
ZYNTEGLO was evaluated in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for TI. Patients were evaluable if they had completed 24 months of follow-up in the phase 3 study, or achieved TI, or won’t achieve TI in the phase 3 study.
Total hemoglobin levels presented about ZYNTEGLO reflect weighted average total hemoglobin during transfusion independence, which is defined as the average across hemoglobin levels taken at each patient visit during the clinical study, factoring in a weighting based on the time duration between study visits.
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
*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.
ZYNTEGLO was evaluated in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for TI. Patients were evaluable if they had completed 24 months of follow-up in the phase 3 study, or achieved TI, or won’t achieve TI in the phase 3 study.
Total hemoglobin levels presented about ZYNTEGLO reflect weighted average total hemoglobin during transfusion independence, which is defined as the average across hemoglobin levels taken at each patient visit during the clinical study, factoring in a weighting based on the time duration between study visits.
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
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