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patisiran   Click here for help

GtoPdb Ligand ID: 13611

Synonyms: ALN-18328 | ALN-TTR02 | Onpattro®
Approved drug
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.
Classification Click here for help
Compound class Nucleic acid
Compound subclass siRNA
Target transthyretin (TTR) mRNA
Approved drug? Yes. UK MHRA (2021)  |  EU EMA (2018)  |  US FDA (2018)
International Nonproprietary Names Click here for help
INN number INN
9793 patisiran
Synonyms Click here for help
ALN-18328 | ALN-TTR02 | Onpattro®
Database Links Click here for help
CAS Registry No. 1420706-45-1 (source: WHO INN record)
ChEMBL Ligand CHEMBL3989987
DrugBank Ligand DB14582
GtoPdb PubChem SID 504705430
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