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Synonyms: ALN-57213 | ALN-AT3 | ALN-AT3SC
Compound class:
Nucleic acid
Comment: Fitusiran is a small interference RNA (siRNA) class agent. It is intended to reduce the antithrombin (SERPINC1) protein level as a mechanism to promote thrombin synthesis and rebalance hemostasis (and prevent bleeds) in patients with hemophilia A or B [4-5].
GalNAc-conjugation is a strategy used to promote delivery of the drug to the liver [2]- 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. |
