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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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Nucleic Acid Sequence  |
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(3'-5')G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT (5'-3')dT-dT-C-A-Um-U-G-G-U-U-C-U-C-A-Um-A-A-G-G-U-A |
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