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Alipogene Tiparvovec

CHEMBL2108304 Phase 4 承認済み Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

The first approved gene therapy in the Western world, designed to treat lipoprotein lipase deficiency (LPLD), an extremely rare inherited disorder that prevents the body from properly breaking down fat in the blood. It uses a modified virus to deliver a functional copy of the LPL gene to muscle cells, reducing the dangerously high triglyceride levels that cause severe, recurrent pancreatitis.

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)

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.

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

The first approved gene therapy in the Western world, designed to treat lipoprotein lipase deficiency (LPLD), an extremely rare inherited disorder that prevents the body from properly breaking down fat in the blood. It uses a modified virus to deliver a functional copy of the LPL gene to muscle cells, reducing the dangerously high triglyceride levels that cause severe, recurrent pancreatitis.

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, Alipogene Tiparvovec 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). CHEMBL2108304. Open-access bioactivity database.

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

医学的免責事項

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.