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revusiran   Click here for help

GtoPdb Ligand ID: 13631

Synonyms: AD-51547 | ALN-TTRSC
Compound class: Nucleic acid
Comment: Revusiran is a double-stranded small interfering ribonucleic acid (siRNA) class ligand. It is proposed to reduce hepatic transthyretin (TTR) production, as a mechanism to treat hereditary transthyretin-mediated (hATTR) amyloidosis-related cardiomyopathy.
Nucleic acid bases are chemically modified to improve stability and GalNAc-conjugation is used to promote delivery of the drug to the liver [1]- this is annotated as 'R1' in the sequence provided here. We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation.
Classification Click here for help
Compound class Nucleic acid
Compound subclass siRNA
Target transthyretin (TTR) mRNA
International Nonproprietary Names Click here for help
INN number INN
9989 revusiran
Synonyms Click here for help
AD-51547 | ALN-TTRSC
Database Links Click here for help
CAS Registry No. 1438322-82-7 (source: WHO INN record)
ChEMBL Ligand CHEMBL4594492
GtoPdb PubChem SID 504705450
Search PubMed clinical trials revusiran
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Search PubMed titles/abstracts revusiran