Angiopoietin-like 3 (ANGPTL3) iRNA composition and method of use thereof

An iRNA composition targets the ANGPTL3 gene to inhibit its expression, addressing the ineffectiveness of current treatments for lipid metabolism disorders by significantly reducing serum lipids and treating conditions like hyperlipidemia and hypertriglyceridemia.

JP2026113714APending Publication Date: 2026-07-07ALNYLAM PHARMACEUTICALS INC

Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
ALNYLAM PHARMACEUTICALS INC
Filing Date
2026-04-15
Publication Date
2026-07-07

AI Technical Summary

Technical Problem

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

Method used

An iRNA composition is developed to induce RNA-induced silencing complex (RISC) cleavage of the ANGPTL3 gene, inhibiting its expression and reducing serum lipid levels by targeting specific nucleotide sequences in the ANGPTL3 gene using double-stranded ribonucleic acid (dsRNA) with modified nucleotides and ligands.

Benefits of technology

The iRNA composition significantly inhibits ANGPTL3 expression by up to 99%, leading to decreased serum lipid, triglyceride, cholesterol, and free fatty acid levels, effectively treating lipid metabolism disorders.

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Abstract

This invention provides an iRNA composition that induces cleavage of the RNA transcript of the ANGPL3 gene via the RNA-induced silencing complex (RISC). [Solution] A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of ANGPTL3, wherein the dsRNA comprises a sense strand and an antisense strand, the sense strand comprises at least 15 consecutive nucleotides that differ from the nucleotide sequence of SEQ ID NO: 1 by three or fewer nucleotides, and the antisense strand comprises at least 15 consecutive nucleotides that differ from the nucleotide sequence of SEQ ID NO: 5 by three or fewer nucleotides.
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