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
See Full Study DesignsTopics to Explore
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
(β0/β0 and non-β0/β0).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
Explore Patient ProfilesMechanism 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
More on MOASafety 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
Read About ZYNTEGLO Safety