Claims for Patent: 12,460,206
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Summary for Patent: 12,460,206
| Title: | PCSK9 iRNA compositions and methods of use thereof |
| Abstract: | The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia. |
| Inventor(s): | Anna Borodovsky, Kallanthottathil G. Rajeev, Kevin Fitzgerald, Maria Frank-Kamenetsky, William Querbes, Martin A. Maier, Klaus Charisse, Satyanarayana KUCHIMANCHI, Muthiah Manoharan, Stuart Milstein |
| Assignee: | Alnylam Pharmaceuticals Inc |
| Application Number: | US17/586,850 |
| Patent Claims: |
1. A pharmaceutical composition comprising a double stranded RNAi agent that inhibits the expression of Proprotein convertase subtilisin kexin 9 (PCSK9) in a cell, comprising an antisense strand consisting of the nucleotide sequence of 5′-asCfsaAfAfAfgCfaAfaAfcAfgGfuCfuagsasa-3′ (SEQ ID NO:1663), and a sense strand consisting of the nucleotide sequence of 5′-csusagacCfuGfudTuugcuuuugu-3′ (SEQ ID NO:1657) and a ligand, wherein the ligand is conjugated to the 3′-end of the nucleotide sequence of the sense strand as shown in the following schematic wherein a, c, g, and u are 2′-O-methyl (2′-OMe) A, C, G, and U, respectively; Af, Cf, Gf, and Uf are 2′-fluoro A, C, G, and U, respectively; s is a phosphorothioate linkage; dT is 2′-deoxythymidine; and X is O, in a sterile aqueous solution. 2. The pharmaceutical composition of claim 1, wherein the composition is for subcutaneous use. |
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