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Vutrisiran

CHEMBL4594511 Phase 4 Approved Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
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Phase
4

Vutrisiran is a GalNAc-conjugated small interfering RNA (siRNA) that targets and mediates RNase-mediated degradation of transthyretin (TTR) mRNA in hepatocytes via the RNAi pathway, reducing production of both mutant and wild-type TTR protein. It is approved for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy in adults, reducing amyloid deposition in peripheral nerves and other organs. The GalNAc conjugate enables targeted hepatocyte delivery via asialoglycoprotein receptor-mediated endocytosis, allowing subcutaneous administration.

Mechanism of Action

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mechanism

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

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.

Frequently Asked Questions

Vutrisiran is a GalNAc-conjugated small interfering RNA (siRNA) that targets and mediates RNase-mediated degradation of transthyretin (TTR) mRNA in hepatocytes via the RNAi pathway, reducing production of both mutant and wild-type TTR protein. It is approved for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy in adults, reducing amyloid deposition in peripheral nerves and other organs. The GalNAc conjugate enables targeted hepatocyte delivery via asialoglycoprotein receptor-mediated endocytosis, allowing subcutaneous administration.

Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.

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

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References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4594511. Open-access bioactivity database.

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

Medical Disclaimer

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.