Help them let go of
their transfusions

ZYNTEGLO® is a one-time gene therapy that gives people with beta-thalassemia the potential to become free from regular red blood cell (RBC) transfusions.1

Explore Efficacy

In two open-label, single-arm studies,
89% (n=32/36; 95% CI: 74, 97) of patients achieved
transfusion independence.*

CI = confidence interval.
*Transfusion independence: a weighted average Hb ≥9 g/dL without any packed red blood cell transfusions for a continuous period of ≥12 months at any time during the study after ZYNTEGLO infusion.1
The phase 3 studies evaluated the efficacy of ZYNTEGLO in 41 patients. Of those 41 patients, 36 were evaluable for transfusion independence.

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Actor portrayals
Beta-thalassemia transfusion independence Beta-thalassemia transfusion independence
89% (n=32/36; 95% CI: 74, 97) of patients
across ages and genotypes achieved transfusion
independence with a median weighted average total
Hb during TI of 11.5 g/dL (min, max: 9.3, 13.7)1*

*Across clinical trials, patient evaluability for transfusion independence (n=36) was defined as patients who have completed their parent study (ie, 24 months of follow-up), achieved transfusion independence, or will not achieve transfusion independence in their parent study.1

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Patient Identification

Patient Identification

ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions.1

Made using the patient’s
own hematopoietic stem cells (HSCs).1

Studied in both adult and
pediatric patients.1

Assessed across all genotypes
00 and non-β00).1

*ZYNTEGLO was studied in patients 4 to 34 years of age. The safety and efficacy in children <4 years of age have not been established; no data are available.1

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Mechanism of Action

Mechanism of Action

ZYNTEGLO can address the underlying genetic cause of beta-thalassemia by adding functional copies of a modified β-globin gene to the patient’s own hematopoietic stem cells (HSCs).1

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Safety Considerations

Safety Considerations

Safety was based on 41 patients treated with ZYNTEGLO in 2 open-label, single-arm clinical studies and 1 long-term follow-up study.1

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