Product Details:

CasNo： 1867157-35-4

Molecular Formula： Chemistry‑GalNAc (ESC‑GalNAc) platform oligonucleotide

Appearance： White to off‑white solid

Packing： Bottle

Throughput： 1KG/Month

Purity： 99%

Vutrisiran

Brand Name: Amvuttra®FDA Access...

Therapeutic class: GalNAc‑conjugated double‑stranded small‑interfering RNA (siRNA); Enhanced Stabilization Chemistry‑GalNAc (ESC‑GalNAc) platform oligonucleotidePMC Molecular weight (sodium salt): 17 290 Da

Mechanism of Action (FDA‑SPL‑aligned professional wording)

Vutrisiran is a chemically‑modified, double‑stranded siRNA covalently conjugated to a tri‑N‑acetylgalactosamine (GalNAc) ligand for hepatocyte‑targeted deliveryFDA Access.... Upon hepatic uptake via asialoglycoprotein receptor‑mediated endocytosis, vutrisiran mediates RNA‑interference‑driven degradation of both wild‑type and mutant transthyretin (TTR) messenger RNA. This suppresses hepatic TTR protein biosynthesis, lowering circulating TTR concentrations. Reduced TTR abundance mitigates misfolded TTR monomer formation and subsequent amyloid fibril deposition in peripheral nerves and cardiac tissue, addressing the root pathogenesis of transthyretin‑mediated amyloidosisAlnylam Ph.... It employs enhanced stabilization chemistries including 2′‑O‑methyl, 2′‑fluoro modifications and phosphorothioate linkages to confer metabolic stability and prolonged pharmacodynamic effect, permitting quarterly subcutaneous dosingPMC.

Indications and Usage (Approved Clinical Label)

Vutrisiran is indicated for adult patients for the treatment of:

1. Polyneuropathy of hereditary transthyretin‑mediated amyloidosis (hATTR‑PN), to slow progression of peripheral nerve impairmentAlnylam Ph....
2. Cardiomyopathy of wild‑type or hereditary transthyretin‑mediated amyloidosis (ATTR‑CM), for reduction of cardiovascular mortality, cardiovascular‑related hospitalizations and urgent heart‑failure visitsAlnylam Ph....

Clinical administration: Subcutaneous injection; recommended dose 25 mg once every 3 months (quarterly)

Research‑Material Disclaimer This oligonucleotide API is supplied For Research Use Only (RUO). It is not manufactured under cGMP conditions for human clinical use. Any human‑directed formulation or subcutaneous administration requires full local regulatory authorization and compliant cGMP production.