Vutrisiran

**Semantic type:** Nucleic Acid, Nucleoside, or Nucleotide|Pharmacologic Substance

**Definition:** A N-acetylgalactosamine (GalNAc)-conjugated small-interfering RNA (siRNA) directed against transthyretin (TTR), that can be used in the treatment of TTR-mediated amyloidosis (ATTR). Upon subcutaneous administration of vutrisiran, the GalNAc moiety targets and binds with high affinity to asialoglycoprotein receptors (ASGPRs) expressed on hepatocytes. Once inside the cells, the siRNAs bind to and silence TTR mRNAs, and inhibit both the translation and expression of the TTR protein. ATTR is caused by mutations in the TTR gene and results in the formation of abnormal amyloid proteins that accumulate in and cause damage to various body organs and tissues.

**Synonyms:** - ALN 65492 - ALN-65492 - ALN-TTRSC02 Free Acid - ALN65492 - VUTRISIRAN