Angiopoietin-like 3(angptl3) irna compostions and methods of use thereof

Double-stranded RNA is used to inhibit ANGPTL3 gene expression, effectively reducing serum lipids and treating lipid metabolic disorders by up to 99% through RNA-induced silencing, addressing the inadequacies of current treatments.

KR103000620B1Active Publication Date: 2026-08-05알닐람파마슈티칼스인코포레이티드
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Patent Information

Application Number
KR1020237023224
Authority / Receiving Office
KR · KR
Patent Type
Patents
Current Assignee / Owner
Priority Date
2012-04-25
Filing Date
2012-06-20
Publication Date
2026-08-05
Estimated Expiration
2032-06-20

AI Technical Summary

Technical Problem

Current treatments for lipid metabolic disorders, such as hyperlipidemia and hypertriglyceridemia, are not always effective, and there is a need for alternative therapies to manage elevated serum lipid levels associated with cardiovascular disease and other pathological conditions.

Method used

The use of double-stranded ribonucleic acid (dsRNA) to inhibit the expression of the ANGPTL3 gene through RNA-induced silencing complex-mediated cleavage, which includes specific sense and antisense strands with complementary regions, optionally modified nucleotides, and conjugated ligands to target and suppress ANGPTL3 expression in cells.

Benefits of technology

The dsRNA effectively suppresses ANGPTL3 expression by up to 99%, leading to significant reductions in serum lipids, triglycerides, cholesterol, and ANGPTL3 protein levels, thereby treating lipid metabolism disorders.

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Abstract

The present invention provides an iRNA composition that causes RNA-induced silencing complex (RISC)-mediated cleavage of the RNA transcript of the ANGPL3 gene to occur. The ANGPL3 gene may be present in cells, e.g., within a subject such as a human. Furthermore, the present invention provides a method of using the iRNA composition of the present invention to treat a subject who suffers from or is prone to lipid metabolism disorders, such as a subject who suffers from or is prone to hyperlipidemia or hypertriglyceridemia, by suppressing the expression of the ANGPL3 gene and / or benefiting from the suppression or reduction of the expression of the ANGPL3 gene.
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