Exagamglogene Autotemcel
Exagamglogene autotemcel is a gene therapy product consisting of CRISPR/Cas9 gene-edited autologous CD34+ cells. It is designed to treat patients with certain hemoglobin disorders by increasing fetal hemoglobin (HbF) production through genetic modification of blood-forming stem cells.
A one-time gene therapy that edits patients' own stem cells to increase fetal hemoglobin production for the treatment of beta-thalassemia and sickle cell disease.
Key facts at a glance
- ATC
- B06AX05
- RxNorm
- 2671667
- Generic
- Exagamglogene Autotemcel
- Class
- atc-b06
- Brands
- Casgevy
- 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
- dose based on body weight
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- dose based on body weight
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- dose based on body weight
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid treatment
exagamglogene-autotemcel is contraindicated during pregnancy due to required conditioning chemotherapy and potential risks to the fetus
not applicable - avoid in pregnancy
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid treatment
defer treatment until after delivery; conditioning regimen is contraindicated during pregnancy
not applicable - avoid in pregnancy
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid treatment
postpone treatment until after childbirth; discuss alternative management of underlying condition during pregnancy
not applicable - avoid in pregnancy
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingavoid if possible
breastfeeding should be avoided during treatment and for some time after infusion due to conditioning chemotherapy and unknown excretion into breast milk
consult with specialist regarding timing of breastfeeding resumption
pediatric_age
adolescents 12 17approved for patients 12 years and older with sickle cell disease
must meet eligibility criteria including adequate organ function; requires thorough evaluation of risks vs benefits in adolescent population
regular monitoring of hematologic parameters, organ function, and potential delayed complications
Use with caution and monitor for adverse effects.
geriatric
elderly 65 plusindividualize based on fitness
eligibility depends on overall health status and organ function; conditioning chemotherapy tolerance must be carefully assessed
comprehensive geriatric assessment recommended; close monitoring for treatment-related complications
Use with caution and monitor for adverse effects.
renal_impairment
mildno specific dose adjustment documented
assess renal function before treatment; monitor renal function during and after treatment
regular serum creatinine and eGFR monitoring
Use with caution and monitor for adverse effects.
renal_impairment
moderate severeuse with caution
careful risk-benefit assessment required; may require modified conditioning or enhanced monitoring
intensive renal function monitoring; consider nephrology consultation
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno specific dose adjustment documented
assess hepatic function before treatment; monitor liver enzymes during treatment
regular liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severeuse with extreme caution
careful evaluation required; may be contraindicated depending on severity; consult with hepatologist
frequent liver function monitoring; may require dose modification of conditioning regimen
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
FDA approval year
FDA approved Exagamglogene Autotemcel (Casgevy) for sickle cell disease
Drug class
Ex vivo CRISPR-Cas9 gene-edited autologous hematopoietic stem cell therapy
Approved indications
Approved for treatment of sickle cell disease and beta-thalassemia
List price
One-time gene therapy treatment cost
Treatment of beta-thalassemia and sickle cell disease in patients who require regular blood transfusions. It is a one-time, personalized gene therapy administered via intravenous infusion of the patient's own edited stem cells.
The therapy involves collecting a patient's CD34+ stem cells, which are then edited using CRISPR/Cas9 technology to reduce expression of BCL11A. After reinfusion, these edited cells engraft in the bone marrow and differentiate into red blood cells that produce increased levels of fetal hemoglobin (HbF), which can substitute for defective adult hemoglobin.
Exagamglogene autotemcel is manufactured specifically for each individual patient using their own cells and cannot be interchanged with other products. As a gene therapy, it is intended for a single administration. Potential risks include infusion reactions, cytopenias, and the requirement for conditioning chemotherapy prior to treatment. Patients should be monitored by healthcare providers experienced in gene therapy and stem cell transplantation.
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
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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