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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  |
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| Compound class | Nucleic acid |
| Compound subclass | siRNA |
| Target | transthyretin (TTR) mRNA |
| International Nonproprietary Names |
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| INN number | INN |
| 9989 | revusiran |
| Synonyms |
| AD-51547 | ALN-TTRSC |
| Database Links |
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| CAS Registry No. | 1438322-82-7 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL4594492 |
| GtoPdb PubChem SID | 504705450 |
| Search PubMed clinical trials | revusiran |
| Search PubMed titles | revusiran |
| Search PubMed titles/abstracts | revusiran |
