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US 12,709,748 B2Granted
Huntingtin (HTT) iRNA agent compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsMangala Meenakshi Soundarapandian, James D. McIninch, Bret Lee Bostwick, Mark K. Schlegel, Adam Castoreno, Jeffrey Zuber
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Abstract
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Huntingtin (HTT) gene, e.g., exon 1 of an HTT gene, as well as methods of inhibiting expression of an HTT gene and methods of treating subjects having an HTT-associated disease or disorder, e.g., Huntington's disease, using such dsRNAi agents and compositions.
Claims29
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Huntingtin (HTT), or a salt thereof,
- wherein the dsRNA agent, or a salt thereof, comprises a sense strand and an antisense strand forming a double stranded region,
- wherein the antisense strand comprises at least 15 contiguous nucleotides from the nucleotide sequence 5′-UAUCAGCUUUUCCAGGGUCGCCG-3′ of SEQ ID NO:
- 4200,
- wherein all of the nucleotide of the sense strand and all of the nucleotides of the antisense strand comprise a nucleotide modification, and
- wherein one or more lipophilic moieties are conjugated to one or more internal positions on the sense strand.
2 more elements
Dependencies are inferred from the claim text.