Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsADAM CASTORENO, ELANE FISHILEVICH, JASON A. GILBERT, CHARALAMBOS KAITTANIS, JAMES D. MCININCH, STUART MILSTEIN, MARK K. SCHLEGEL
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Abstract
The disclosure relates to double-stranded ribonucleic acid (dsRNA) agents and compositions targeting the ATXN3 gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an ATXN3-associated disease or disorder, such as SCA3, using such dsRNA agents and compositions.
Claims112
A double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of ATXN3, 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 or 1 mismatches, of 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 or 1 mismatches, of the corresponding portion of nucleotide sequence of SEQ ID NO:
- 2 such that the sense strand is complementary to the at least 15 contiguous nucleotides in the antisense strand, wherein substitution of a thymidine base for a uridine base is not considered a mismatch.
A double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of ATXN3, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein the antisense strand comprises a nucleotide sequence of an antisense sequence listed in any one of Tables 2, 5, 7A, 10, 11, or 14, and the sense strand comprises a nucleotide sequence of a sense sequence listed in any one of Tables 2, 5, 7A, 10, 11, or 14 that corresponds to the antisense sequence.
Dependencies are inferred from the claim text.