ACOX1 lowering antisense oligonucleotide compositions and methods of use thereof

WO2026152036A1PCT designated stage Publication Date: 2026-07-16WASHINGTON UNIV IN SAINT LOUIS
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Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
WASHINGTON UNIV IN SAINT LOUIS
Filing Date
2026-01-09
Publication Date
2026-07-16

AI Technical Summary

Technical Problem

There is a need for effective treatments for ACOX1-associated conditions, particularly ACOX1-associated neurodegenerative disorders such as Mitchell Syndrome, which are caused by hyperactive ACOX1 protein leading to ROS accumulation and cell death, with no current cure available.

Method used

The use of ACOX1 mRNA targeting antisense oligonucleotides (ASOs) to decrease ACOX1 protein levels and enzymatic activity, administered at concentrations ranging from 1 nM to 1 pM, effectively reducing ACOX1 expression in human cells and tissues.

Benefits of technology

The ASOs significantly decrease ACOX1 protein abundance and enzymatic activity, showing promise in treating ACOX1-associated conditions by lowering ACOX1 mRNA levels and improving metabolic outcomes in human cell lines and patient-derived cells.

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Abstract

Compositions and methods for treating a neurological disorder are provided. Exemplary compositions include at least one antisense oligonucleotide (ASO) as disclosed herein, wherein the at least one ASO targets an ACOX1 mRNA transcript. Exemplary methods include treating an ACOX1-associated condition in a subject in need thereof and lowering ACOX1 expression in cells, including human cells, by administering an exemplary composition. An ACOX1-associated condition includes at least one of obesity, an ACOX1-associated neurodegenerative disorder, an ACOX1 mutation syndrome, an ACOX1 gain-of-function mutation, Mitchell syndrome, an ACOX1-associated peroxisomal disorder, a peroxisomal brain disorder, a peroxisomal peripheral nervous system disorder, a peroxisomal central nervous system disorder, and an increased peroxisome beta oxidation associated disease.
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