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US 2026/0028628 A1Published application
Compositions and methods for inhibition of expression of inhibin subunit beta e (inhbe) genes
AssigneeBasecure Therapeutics LLC
InventorsJing YANG, Kevin J. KIM, Saul Martinez MONTERO
Life of this patent
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Abstract
The disclosure relates to double-stranded ribonucleic acid (dsRNA) targeting an INHBE gene, and methods of using the dsRNA to inhibit expression of INHBE.
Claims18
1-81. (canceled)
A double-stranded ribonucleic acid (dsRNA), wherein the dsRNA comprises a sense strand and an antisense strand each 18 to 25 nucleotides in length, wherein:
- (a) the antisense strand comprises a nucleobase sequence of at least 15 contiguous nucleotides ofAAGACGGCAGAAUGGAAAGAGGC (SEQ ID NO:
- 217);
- and
- (b) the sense strand comprises a nucleobase sequence of at least 15 contiguous nucleotides of CUCUUUCCAUUCUGCCGUCUU (SEQ ID NO:
- 71);
- wherein at least one nucleotide of the dsRNA is a modified nucleotide selected from a 2′-O-methyl modified nucleotide or a 2′-fluoro modified nucleotide;
- wherein at least one internucleoside linkage is a phosphorothioate linkage;
- and
- wherein the dsRNA further comprises a ligand or targeting moiety that comprises at least one N-acetyl-galactosamine (GalNAc).
6 more elements
A double-stranded ribonucleic acid (dsRNA), wherein the dsRNA comprises an antisense strand of 23 nucleotides in length and a sense strand of 21 nucleotides in length, wherein:
- (a) the antisense strand comprises a nucleobase sequence of 17 contiguous nucleotides ofAAGACGGCAGAAUGGAAAGAGGC (SEQ ID NO:
- 217);
- and
- (b) the sense strand comprises a nucleobase sequence of 17 contiguous nucleotides of CUCUUUCCAUUCUGCCGUCUU (SEQ ID NO:
- 71);
- wherein the antisense strand has a 3′ end nucleotide overhang compared to the sense strand;
- wherein the antisense strand and the sense strand each comprise at least one modified nucleotide selected from a 2′-O-methyl modified nucleotide or a 2′-fluoro modified nucleotide;
- wherein the antisense strand and the sense strand each comprise at least one phosphorothioate linkage;
- and
- wherein the dsRNA further comprises a ligand or targeting moiety that comprises at least one N-acetyl-galactosamine (GalNAc), wherein the ligand or targeting moiety is conjugated to the 5′ end of the sense strand of the dsRNA.
7 more elements
Dependencies are inferred from the claim text.