TMPRSS6 iRNA compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsJames Butler, Martin Maier, Kevin Fitzgerald, Shannon Fishman, Donald Foster, Vasant Jadhav, Stuart Milstein
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Abstract
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
Claims14
A double stranded RNAi agent for inhibiting expression of TMPRSS6 in a cell, wherein said double stranded RNAi agent comprises a sense strand and an antisense strand forming a duplex, wherein the sense strand comprises any one of the modified nucleotide sequences selected from the group consisting of SEQ ID NOS:
- 62-86, 112-127, and 144-147, and the antisense strand comprises any one of the modified nucleotide sequences selected from the group consisting of SEQ ID NOS:
- 87-111, 128-143, and 148-151, and wherein the sense strand is 21 nucleotides in length and the antisense strand is 23 nucleotides in length.
Dependencies are inferred from the claim text.