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Synonyms: ALN-18328 | ALN-TTR02 | Onpattro®
patisiran is an approved drug (EMA & EMA & FDA (2018), UK MHRA (2021))
Compound class:
Nucleic acid
Comment: Patisiran (ALN-TTR02) is a double-stranded small interfering RNA (siRNA) class drug [2]. It reduces hepatic production of mutated transthyretin protein, as a mechanism to treat hereditary transthyretin amyloidosis with polyneuropathy [1,3-4]. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
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Classification  |
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| Compound class | Nucleic acid |
| Compound subclass | siRNA |
| Target | transthyretin (TTR) mRNA |
| Approved drug? | Yes. FDA (2018) | EMA (2018) | UK MHRA (2021) |
| International Nonproprietary Names |
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| INN number | INN |
| 9793 | patisiran |
| Synonyms |
| ALN-18328 | ALN-TTR02 | Onpattro® |
| Database Links |
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| CAS Registry No. | 1420706-45-1 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL3989987 |
| DrugBank Ligand | DB14582 |
| GtoPdb PubChem SID | 504705430 |
| Search PubMed clinical trials | patisiran |
| Search PubMed titles | patisiran |
| Search PubMed titles/abstracts | patisiran |
