US 2024/0352464 A1 · Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof

Abstract

Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses thereof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2′-O-methyl nucleotides and 2′-fluoro nucleotides. Further disclosed herein are siNA molecules comprising additional modification including a phosphorylation blocker, conjugated moiety, or 5′-stabilized end cap. The siNA molecules may reduce or inhibit the production of hydroxysteroid dehydrogenase.

Claims20

Dependent claims

A double-stranded short interfering nucleic acid (siNA) molecule comprising:

(a) a sense strand comprising a nucleotide sequence that is at least about 60%, 65%, 70%, 75%, 80%, 85%, 90%, 95%, or 100% identical to a nucleotide sequence of any one of SEQ ID NOs:
1-25, 68-75, and 87-94;
and/or
(b) an antisense strand comprising a nucleotide sequence that is at least about 60%, 65%, 70%, 75%, 80%, 85%, 90%, 95%, or 100% identical to a nucleotide sequence of any one of SEQ ID NOs:
26-67, 76-86, and 95-97,
wherein the siNA molecule downregulates expression of a hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) gene.

3 more elements

Dependencies are inferred from the claim text.

Facts

Application

18/599,052Filed 7 Mar 2024

Priority

7 Mar 2023

Published

24 Oct 2024

Term ends

Not estimatedThe source records don’t support an estimate.

Classification10 codes

C12N 15/1137
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming…: against enzymes
C12Y 101/01062
With NAD+ or NADP+ as acceptor: 17Beta-estradiol 17-dehydrogenase
C12N 2310/14
Type of nucleic acid: interfering nucleic acids [NA]
C12N 2310/3125
Phosphonates: methylphosphonates

Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof

Life of this patent

Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof

Life of this patent

Abstract

Claims20