Mucin 5b (muc5b) irna compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsLeila Noetzli, Margaret Parker, James D. McIninch, Mark K. Schlegel, Adam Castoreno, Jeffrey Zuber
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Abstract
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the mucin 5B (MUC5B) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a MUC5B gene and to methods of treating or preventing a MUC5B-associated disease, such as a lung disease, e.g., cystic fibrosis, chronic obstructive pulmonary disease (COPD), and/or pulmonary fibrosis, e.g., idiopathic pulmonary fibrosis (IPF), in a subject.
Claims40
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Mucin 5B (MUC5B) in a cell,
- (a) wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region,
- wherein the sense strand comprises a nucleotide sequence comprising at least 15 contiguous nucleotides, with 0, 1, 2, or 3 mismatches, of a portion of the nucleotide sequence of SEQ ID NO:1, or a nucleotide sequence having at least 90% nucleotide sequence identity to a portion of the nucleotide sequence of SEQ ID NO:1, and the antisense strand comprises a nucleotide sequence comprising at least 15 contiguous nucleotides, with 0, 1, 2, or 3 mismatches, of the corresponding portion of the nucleotide sequence of SEQ ID NO:6, or a nucleotide sequence having at least 90% nucleotide sequence identity to a portion of the nucleotide sequence of SEQ ID NO:6;
- and
- wherein the sense strand or the antisense strand is conjugated to one or more lipophilic moieties;
- or
- (b) wherein the antisense strand comprises a region complementary to part of an mRNA encoding a MUC5B gene (SEQ ID NO:1), wherein each strand independently is 14 to 30 nucleotides in length;
- and wherein the sense strand or the antisense strand is conjugated to one or more lipophilic moieties;
- or
- (c) wherein the antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense nucleotide sequences in any one of Tables 2-7, 9, and 10, wherein each strand independently is 14 to 30 nucleotides in length;
- and wherein the sense strand or the antisense strand is conjugated to one or more lipophilic moieties.
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Dependencies are inferred from the claim text.