よくある質問

What is Exagamglogene Autotemcel used for?

This gene editing therapy uses CRISPR technology on a patient's own stem cells to reactivate fetal hemoglobin production, providing a potentially curative treatment for sickle cell disease and transfusion-dependent beta-thalassemia. The edited cells are returned to the patient after conditioning.

Is Exagamglogene Autotemcel an approved drug?

Yes, Exagamglogene Autotemcel is an approved drug. It has reached clinical phase 4. It is classified as a Gene.

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References & Data Sources

• ChEMBL — European Bioinformatics Institute (EBI). CHEMBL5095474. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.