Atidarsagene Autotemcel
An ex vivo gene therapy in which a patient's own stem cells are genetically modified to insert a functional copy of the ARSA gene, and then reinfused to produce a missing enzyme in patients with metachromatic leukodystrophy.
作用機序
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
HBD / HBA
- / -
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
よくある質問
An ex vivo gene therapy in which a patient's own stem cells are genetically modified to insert a functional copy of the ARSA gene, and then reinfused to produce a missing enzyme in patients with metachromatic leukodystrophy.
Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.
Yes, Atidarsagene Autotemcel is an approved drug. It has reached clinical phase 4. It is classified as a Gene.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4802262. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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