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Patisiran Sodium

CHEMBL4297536 Phase 4 Zugelassen Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A sodium salt form of patisiran, an RNA interference (RNAi) therapy that silences the TTR gene in the liver, reducing production of misfolded transthyretin protein that causes hereditary transthyretin-mediated amyloidosis. Reducing TTR levels slows the progression of nerve and heart damage from this rare genetic disease.

Wirkmechanismus

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mechanismus

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

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

A sodium salt form of patisiran, an RNA interference (RNAi) therapy that silences the TTR gene in the liver, reducing production of misfolded transthyretin protein that causes hereditary transthyretin-mediated amyloidosis. Reducing TTR levels slows the progression of nerve and heart damage from this rare genetic disease.

Stabilizes the transthyretin (TTR) protein tetramer, preventing its dissociation into monomers that misfold and aggregate into amyloid fibrils in cardiac tissue.

Yes, Patisiran Sodium is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.

{# 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). CHEMBL4297536. 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.

Data sources: ChEMBL, PubChem, DailyMed.