Tool and method for disaggregation of polyq strech containing proteins

WO2025068459A3PCT designated stage expired Publication Date: 2025-05-08UNIVERSITY OF COLOGNE
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Patent Information

Application Number
PCT/EP2024/077215
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-02-23
Filing Date
2024-09-27
Publication Date
2025-05-08

AI Technical Summary

Technical Problem

Current treatments for neurodegenerative diseases caused by polyQ-expanded proteins, such as Huntington's disease, are inadequate as they either suppress both healthy and mutant protein production or fail to reach the brain effectively, leading to side effects and limited efficacy.

Method used

Development of a protein with antiaggregating and disaggregating activity, specifically a plant-derived chloroplast stromal processing peptidase (SPP) or its variants, which can target and reduce the aggregation of polyQ-expanded proteins without affecting healthy protein expression.

Benefits of technology

The use of SPP or its variants effectively reduces the aggregation of polyQ-expanded proteins, thereby decreasing neurotoxicity and providing a potential therapeutic approach for neurodegenerative diseases without the side effects associated with existing treatments.

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Abstract

The present invention provides an isolated protein exhibiting an antiaggregating activity and / or disaggregating activity toward a target protein comprising an extended polyQ stretch. The protein comprises a Zn2+-binding region, wherein the conserved motif is HxxEHx75-80E and x is any amino acid. The nucleic acid construct encoding said protein as well as the corresponding mRNA sequence are also provided. The protein, the nucleic acid construct or mRNA sequence are for use in a method for prevention or treatment of a neurodegenerative disease that is caused by aggregates comprising at least one target protein and / or by the mRNA encoding for said target protein, wherein the target protein causes e.g. Huntington's disease or Machado-Joseph disease.
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Citation Information

Patent Citations

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  • Novel nucleic acids and polypeptides

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  • Use of amylase or maltose to treat or prevent neurodegeneration

    WO2022040022A1