US 2026/0209772 A1 · NOVEL RNA THERAPEUTICS AND USES THEREOF

Abstract

The present disclosure relates to novel therapeutic compounds, known as RNAi agents, that decrease expression of the HMGCR receptor (expressed by the HMGCR gene), thereby decreasing expression of mRNA and protein expression. Such RNAi agents are useful in the treatment of diseases and disorders involving the regulation of HMGCR expression and function, such as diseases and disorders that are risk factors for ASCVD (such as, dyslipidemia, such as hypercholesteremia).

Claims37

Dependent claims

An RNAi agent for reducing HMGCR gene expression, wherein the RNAi agent comprises a delivery moiety of Formula I conjugated to R, wherein R is a double stranded RNA (dsRNA) comprising an antisense strand and a sense strand:

wherein R is conjugated to connection point E of Formula I, optionally via a linker, wherein the sense strand and the antisense strand form a duplex region, and wherein the antisense strand comprises a region of complementarity to a HMGCR mRNA target sequence of SEQ ID NO:
1, and wherein the sense and antisense strand each optionally comprise one or more modified nucleotides and one or more modified internucleotide linkages.

Dependencies are inferred from the claim text.

Facts

Application

19/141,216Filed 22 Dec 2023

Priority

23 Dec 2022

Published

23 Jul 2026

Term ends

Not estimatedThe source records don’t support an estimate.

Classification18 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
A61K 31/712
Compounds having three or more nucleosides or nucleotides: nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or…
A61K 31/7125
Compounds having three or more nucleosides or nucleotides: nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other…
A61P 3/06
Drugs for disorders of the metabolism: antihyperlipidemics

Novel rna therapeutics and uses thereof

Life of this patent

Novel rna therapeutics and uses thereof

Life of this patent

Abstract

Claims37