SERUM AMYLOID P COMPONENT (APCS) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsKevin Fitzgerald, Gregory Hinkle
Life of this patent
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Abstract
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the serum amyloid P component (APCS) gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of an APCS gene and to treat subjects having an APCS-associated disease, e.g., amyloidosis, Alzheimer's disease or coronary atherosclerotic heart disease.
Claims47
A double stranded ribonucleic acid (RNAi) agent for inhibiting expression of a serum amyloid P component (APCS) gene, comprising a sense strand and an antisense strand, wherein said sense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:1, and said antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:5.
A double stranded ribonucleic acid (RNAi) agent for inhibiting expression of a serum amyloid P component (APCS) gene, comprising a sense strand and an antisense strand, wherein the antisense strand comprises a region of complementarity comprising at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense sequences listed in any one of Tables 3A, 3B, 4A, 4B, 6, and 7.
A double stranded ribonucleic acid (RNAi) agent for inhibiting expression of a serum amyloid P component (APCS) gene, comprising a sense strand and an antisense strand, wherein said antisense strand comprises a region complementary to part of an mRNA encoding SAP, wherein each strand is about 14 to about 30 nucleotides in length, wherein said sense strand is represented by formula (I):
- sense:
- (I)
- 5′ np-Na-(XXX)i-Nb-YYY-Nb-(ZZZ)j-Na-nq 3′
- wherein:
- i and j are each independently 0 or 1;
- p and q are each independently 0-6;
- Na represents an oligonucleotide sequence comprising 0-25 nucleotides which are either modified or unmodified or combinations thereof;
- Nb represents an oligonucleotide sequence comprising 0-10 nucleotides which are either modified or unmodified or combinations thereof;
- each np and nq, each of which may or may not be present, independently represents an overhang nucleotide;
- XXX, YYY and ZZZ each independently represent one motif of three identical modifications on three consecutive nucleotides;
- modifications on Nb differ from the modification on Y;
- and
- wherein the sense strand is conjugated to at least one ligand.
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Dependencies are inferred from the claim text.