Alipogene Tiparvovec
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.
Wirkmechanismus
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.
Häufig gestellte Fragen
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
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL2108304. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
Medizinischer Haftungsausschluss
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.
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