Snca-targeting sirna compositions for treating snca-associated disease
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsLan Thi Hoang Dang, Mark K. Schlegel, Joseph Barry, Tuyen M. Nguyen, Adam Castoreno, Kawai So
Life of this patent
Estimating the term…
Abstract
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, particularly in a CNS tissue, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy (MSA), Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.
Claims30
A double stranded ribonucleic acid (dsRNA) agent, or a pharmaceutically acceptable salt thereof, comprising a sense strand and an antisense strand forming a double stranded region, wherein the nucleotide sequence of the antisense strand differs by no more than 4 modified or unmodified nucleotides from the nucleotide sequence 5′-VPusdCscadAcdAuuugdTcAfcuugcucsusu-3′ of SEQ ID NO:64,
- wherein a, g, c and u are 2-O-methyl (2-OMe) A, G, C and U;
- Af, Gf, and Cf are 2-fluoro (2-F) A, G, and C;
- dA, dT, and dC are 2-deoxy A, T, and C;
- (Ahd) is 2′-O-hexadecyl adenosine-3′-phosphate;
- VP is Vinyl-phosphonate;
- and s is a phosphorothioate linkage.
A sodium salt of a double stranded ribonucleic acid (dsRNA) agent comprising a sense strand and an antisense strand forming a double stranded region, wherein the sense strand consists of the nucleotide sequence 5′-gsasgca(Ahd)guGfAfCfaaauguugsgsa-3′ of SEQ ID NO:21 and the antisense strand consists of the nucleotide sequence 5′-VPusdCscadAcdAuuugdTcAfcuugcucsusu-3′ of SEQ ID NO:64, wherein a, g, c and u are 2-O-methyl (2-OMe) A, G, C and U;
- Af, Gf, and Cf are 2-fluoro (2-F) A, G, and C;
- dA, dT, and dC are 2-deoxy A, T, and C;
- (Ahd) is 2′-O-hexadecyl adenosine-3′-phosphate;
- VP is Vinyl-phosphonate;
- and s is a phosphorothioate linkage.
Dependencies are inferred from the claim text.