RNAi agent for inhibiting the expression of superoxide dismutase 1 (SOD1), composition thereof, and method of use

RNAi agents targeting SOD1 gene expression in ALS offer a safer and more effective treatment for ALS by reducing SOD1 levels, addressing the limitations of current therapies.

JP2025522433APending Publication Date: 2025-07-15ARROWHEAD PHARMACEUTICALS INC
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Patent Information

Application Number
JP2024573511
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-04-11
Filing Date
2023-06-14
Publication Date
2025-07-15

AI Technical Summary

Technical Problem

Current treatments for amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations have limited effectiveness and are associated with adverse events, necessitating a safer and more effective method to inhibit SOD1 gene expression.

Method used

Development of RNA interference (RNAi) agents, such as chemically modified small interfering RNAs (siRNAs), specifically designed to target and inhibit SOD1 gene expression in central nervous system cells, using pharmacokinetic and pharmacodynamic modulators for efficient delivery.

Benefits of technology

The RNAi agents effectively reduce SOD1 expression, potentially slowing ALS progression with fewer adverse events and providing a safer therapeutic option.

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Abstract

An RNAi agent, a composition containing the RNAi agent, and a method for inhibiting the superoxide dismutase 1 (SOD1) gene are described. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of the SOD1 gene. Pharmaceutical compositions containing one or more SOD1 RNAi agents, optionally together with one or more additional therapeutic agents, are also described. In vivo delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissues results in inhibition of SOD1 gene expression and reduction of SOD1 activity, and can provide a therapeutic effect in the treatment of various diseases, including amyotrophic lateral sclerosis (ALS), to subjects including human subjects.
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