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Atidarsagene Autotemcel

CHEMBL4802262 Phase 4 Đã phê duyệt Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

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.

Cơ chế tác dụng

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)

Cơ chế

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.

Câu hỏi thường gặp

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 section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

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.

Tuyên bố miễn trách y tế

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.