Frequently Asked Questions (FAQs)
Find answers to commonly asked questions about ZYNTEGLO®.
& Gene Therapy
Yes, ZYNTEGLO uses a patient’s own blood stem cells to treat beta-thalassemia.See How ZYNTEGLO Works
ZYNTEGLO works by adding working copies of the beta-globin gene to a patient’s own blood stem cells to treat beta-thalassemia.Learn More About How ZYNTEGLO Works
You will need to speak to your doctor about getting started on ZYNTEGLO. In the meantime, you can find resources at my bluebird support, where you can connect with a Patient Navigator to help support you in the treatment journey.Go to my bluebird support
Studies & Approval
ZYNTEGLO treats people with beta-thalassemia who require regular red blood cell transfusions. This includes pediatrics & adults and all genotypes—β0/β0, where no beta-globin is produced, and non-β0/β0, where a reduced amount of beta-globin is produced. To find out if you are eligible for ZYNTEGLO, talk to your doctor. The safety and efficacy of ZYNTEGLO was studied in clinical trials that enrolled patients between the ages of 4 and 34 years old.See ZYNTEGLO Clinical Results
ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.See ZYNTEGLO Clinical Results
Efficacy & Safety
In two phase 3 studies, about 9 out of 10 (89%; 32/36) patients treated with ZYNTEGLO stopped transfusions and had a median total hemoglobin level of 11.5 g/dL .*†‡
*Weighted Average Total Hb during TI.
†Transfusion independence means that patients achieved a weighted average hemoglobin of 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 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.