Betibeglogene Autotemcel
Betibeglogene autotemcel (ZYNTEGLO) is a one-time gene therapy for transfusion-dependent beta-thalassemia in adult and pediatric patients. It works by modifying the patient's own stem cells to add functional copies of the beta-globin gene, allowing them to produce normal red blood cells and potentially eliminate the need for regular transfusions.
A gene therapy that modifies a patient's own stem cells to treat beta-thalassemia by enabling production of functional hemoglobin.
Key facts at a glance
- ATC
- B06AX02
- RxNorm
- 2610349
- Generic
- Betibeglogene Autotemcel
- Class
- atc-b06
- Brands
- Zynteglo
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 2 × 10^6 CD34+ cells/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2 × 10^6 CD34+ cells/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimestercontraindicated
avoid use during pregnancy; requires myeloablative conditioning with busulfan which is teratogenic
not applicable - avoid in pregnancy
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimestercontraindicated
avoid use during pregnancy; defer gene therapy until postpartum period
not applicable - avoid in pregnancy
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingnot established
discontinue breastfeeding before starting treatment; wait until drug cleared from system
no data on excretion in breast milk
pediatric_age
adolescents 12 to 17standard dosing
approved for patients 12 years and older with transfusion-dependent beta-thalassemia
standard monitoring including hematologic assessments, vector copy number monitoring
pediatric_age
children under 12not established
not approved for patients under 12 years; safety and efficacy not established
limited data available
geriatric
elderly 65 and olderindividual assessment required
limited data in patients over 65; evaluate overall health status and comorbidities before treatment
comprehensive geriatric assessment recommended
Use with caution and monitor for adverse effects.
renal_impairment
severe renal impairmentmonitor closely
busulfan (conditioning agent) is cleared renally; dose adjustment may be required for severe impairment
monitor renal function and busulfan levels if available
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severe hepatic impairmentindividual assessment required
no specific studies; evaluate hepatic function and risk of veno-occlusive disease
monitor liver function tests closely
Use with caution and monitor for adverse effects.
Statistics and clinical data
Therapeutic class
Gene therapy product classification
Approved indication
Therapeutic indication for betibeglogene autotemcel
FDA approval year
FDA approval date for Zynteglo
Dosing regimen
Administration schedule
Treatment of transfusion-dependent beta-thalassemia in adult and pediatric patients who require regular red blood cell transfusions. This is a one-time therapy intended to reduce or eliminate transfusion requirements.
The therapy involves collecting the patient's CD34+ hematopoietic stem cells and using a lentiviral vector (BB305) to add functional copies of a modified beta-globin gene. These modified cells are then reinfused back into the patient, where they engraft in the bone marrow and differentiate to produce red blood cells containing functional beta-globin (hemoglobin).
This is an autologous therapy using the patient's own cells. It requires myeloablative conditioning prior to infusion. Potential risks include neutrophil and platelet engraftment failure, delayed platelet engraftment, and risk of hematologic malignancy. Patients should be monitored long-term. Discuss risks and benefits thoroughly with a qualified treatment center experienced in gene therapy.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
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 July 26, 2026. · Read our editorial policy