Treatment for alpha-1 antitrypsin deficiency (AATD)

Low-dose, low-frequency RNAi therapy targeting hepatocytes effectively inhibits AAT gene expression to halt liver disease progression in AATD patients, addressing the accumulation of misfolded Z-AAT protein and reducing liver damage.

JP7864889B2Active Publication Date: 2026-05-25ARROWHEAD PHARMACEUTICALS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Patents
Current Assignee / Owner
Filing Date
2025-03-12
Publication Date
2026-05-25

AI Technical Summary

Technical Problem

Current treatments for alpha-1 antitrypsin deficiency (AATD) do not effectively address the accumulation of misfolded Z-AAT protein in hepatocytes, leading to liver disease progression and lack a clinically approved therapy to prevent or halt liver damage in AATD patients.

Method used

Administration of a low-dose, low-frequency RNA interference (RNAi) agent, such as ADS-001, which inhibits the expression of the alpha-1 antitrypsin gene, targeting hepatocytes to reduce the accumulation of mutant protein and administered subcutaneously at varying intervals.

Benefits of technology

The RNAi agent slows or halts liver disease progression, including fibrosis and cirrhosis, by reducing AAT gene expression, thereby enabling tissue repair and reducing liver-related complications in AATD patients.

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Abstract

To provide methods for treatment of alpha-1 antitrypsin deficiency (AATD) in a human subject.SOLUTION: Described are methods for treating alpha-1 antitrypsin deficiency (AATD) in a human patient in need of treatment, using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents are administered to a human patient in need thereof for treating liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.SELECTED DRAWING: Figure 3
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Citation Information

Patent Citations

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