Betibeglogene Autotemcel
Zynteglo is a one-time gene therapy for beta-thalassemia that uses the patient's own modified stem cells to enable the body to produce functional beta-globin, reducing or eliminating the need for regular blood transfusions. It is indicated for adult and pediatric patients with transfusion-dependent beta-thalassemia who meet specific eligibility criteria.
Zynteglo is a one-time gene therapy for beta-thalassemia that modifies the patient's own stem cells to enable production of healthy red blood cells, reducing transfusion dependence.
Key facts at a glance
- RxNorm
- 2610355
- Generic
- Zynteglo
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
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Zynteglo is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
This gene therapy involves collecting the patient's own hematopoietic stem cells and genetically modifying them in the laboratory to add a functional copy of the beta-globin gene. The modified cells are then infused back into the patient after chemotherapy conditioning, where they engraft in the bone marrow and produce functional beta-globin, leading to improved hemoglobin levels.
Zynteglo is administered as a single one-time infusion. Patients should be monitored for potential side effects including cytopenias, hepatic veno-occlusive disease, and insertion oncogenesis risk. It is contraindicated in patients with significant comorbidities. Pregnancy and breastfeeding should be avoided after treatment. Patients should have lifelong monitoring per the approved registry.
Official drug label
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This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 27, 2026. · Read our editorial policy