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Synonyms: AD-65585 | ALN-65585 | ALN-G01 | Oxlumo®
lumasiran is an approved drug (EMA, FDA & UK MHRA (2020))
Compound class:
Nucleic acid
Comment: Lumasiran is a double-stranded small interfering ribonucleic acid (siRNA) drug. It targets HAO1 mRNA as a mechanism to reduce the hepatic oxalate overproduction that is responsible for disease pathology in patients with primary hyperoxaluria type 1 (PH1) [1]. 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 | hydroxyacid oxidase 1 (HAO1) |
| Approved drug? | Yes. FDA (2020) | EMA (2020) | UK MHRA (2020) |
| International Nonproprietary Names |
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| INN number | INN |
| 10684 | lumasiran |
| Synonyms |
| AD-65585 | ALN-65585 | ALN-G01 | Oxlumo® |
| Database Links |
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| CAS Registry No. | 1834610-13-7 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL4297779 |
| DrugBank Ligand | DB15935 |
| GtoPdb PubChem SID | 504705432 |
| Search PubMed clinical trials | lumasiran |
| Search PubMed titles | lumasiran |
| Search PubMed titles/abstracts | lumasiran |
