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US 10,125,364 B2Granted
Compositions and methods for inhibiting expression of the ALAS1 gene
AssigneesAlynylam Pharmaceuticals, Inc., Icahn School of Medicine at Mount Sinai
InventorsBrian Bettencourt, Kevin Fitzgerald, William Querbes, Robert J. Desnick, Makiko Yasuda
Life of this patent
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Abstract
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.
Claims36
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, comprising:
- (i) an antisense strand that comprises the sequence of SEQ ID NO:
- 1296;
- (ii) a sense strand comprising at least 15 contiguous nucleotides from SEQ ID NO:
- 1295;
- and
- (iii) a duplex region of 15-30 base pairs in length.
3 more elements
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, comprising:
- (i) an antisense strand that comprises the sequence of SEQ ID NO:
- 1296;
- (ii) a sense strand comprising at least 15 contiguous nucleotides from SEQ ID NO:
- 1295;
- (iii) a duplex region of 15-30 base pairs in length;
- (iv) one strand comprising a 3′ overhang of at least 1 nucleotide;
- (v) one end that is blunt-ended;
- and
- (vi) at least one modified nucleotide chosen from a 2′-O-methyl modified nucleotide, 2′-fluoro modified nucleotide, or both.
6 more elements
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, wherein the dsRNA comprises:
- (i) a sense strand comprising at least 15 contiguous nucleotides from SEQ ID NO:
- 1295;
- (ii) an antisense strand complementary to at least nucleotides 871-889 of SEQ ID NO:
- 1;
- and
- (iii) a ligand comprising one or more N-acetylgalactosamine (GalNAc) derivatives.
3 more elements
Dependencies are inferred from the claim text.