Methods for treating ANGPTL3-associated diseases and disorders

The ANGPTL3 RNAi drug substance effectively treats ANGPTL3-associated diseases by inhibiting gene expression, achieving substantial reductions in triglycerides and cholesterol levels with a favorable safety profile, addressing the limitations of current treatments.

JP2025527670APending Publication Date: 2025-08-22ARROWHEAD PHARMACEUTICALS INC
View PDF 0 Cites 0 Cited by

Patent Information

Application Number
JP2025511500
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-06-08
Filing Date
2023-08-21
Publication Date
2025-08-22

AI Technical Summary

Technical Problem

Current treatments for ANGPTL3-associated diseases, such as hypertriglyceridemia and atherosclerotic cardiovascular disease, are ineffective in significantly reducing triglycerides and cholesterol levels and can cause liver fat accumulation and enzyme elevations, while antibody approaches require inconvenient monthly infusions.

Method used

Administration of a pharmaceutical composition containing an ANGPTL3 RNAi drug substance, such as ADS-003, which inhibits ANGPTL3 gene expression, providing sustained reductions in triglycerides and cholesterol levels without liver fat or enzyme elevation, administered subcutaneously at varying intervals.

Benefits of technology

The ANGPTL3 RNAi drug substance achieves significant reductions in triglycerides, LDL-C, and cholesterol levels with a favorable safety profile, maintaining liver health and avoiding liver fat or enzyme elevations, effectively treating ANGPTL3-associated diseases.

✦ Generated by Eureka AI based on patent content.

Smart Images

  • Figure 2025527670000028
    Figure 2025527670000028
  • Figure 2025527670000029
    Figure 2025527670000029
  • Figure 2025527670000030
    Figure 2025527670000030
Patent Text Reader

Abstract

Methods are described for treating diseases and disorders that may be mediated in part by decreased ANGPTL3 gene expression in a human subject in need thereof using pharmaceutical compositions comprising an ANGPTL3 RNAi agent. The disclosed pharmaceutical compositions comprising an ANGPTL3 RNAi agent, when administered to a human subject in need of treatment according to the methods disclosed herein, treat diseases and disorders associated with elevated triglyceride (TG) levels and / or elevated low-density lipoprotein cholesterol (LDL-C), such as hypertriglyceridemia (including severe hypertriglyceridemia (SHTG)), homozygous familial hypercholesterolemia (HoFH), hypertriglyceridemia-induced pancreatitis, metabolic syndrome, obesity, hyperlipidemia, mixed dyslipidemia, lipid and / or cholesterol metabolism disorders, atherosclerosis, cardiovascular disease, type II diabetes, coronary artery disease, non-alcoholic steatohepatitis, non-alcoholic fatty liver disease, heterozygous familial hypercholesterolemia (HeFH), statin-resistant hypercholesterolemia, other dyslipidemias, and other metabolic-related disorders and diseases.
Need to check novelty before this filing date? Find Prior Art