Mettl3-mettl14-complex inhibitors for the treatment of neuromuscular degenerative pathologies caused by or linked to mutations in the FUS gene

By inhibiting the METTL3-METTL14 complex, the treatment of neuromuscular degenerative disorders, such as ALS, effectively reduces pathological stress granule formation and promotes their dissolution, addressing the limitations of current treatments and improving disease management.

WO2025088514A1 Publication Date: 2025-05-01UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA +1
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Patent Information

Application Number
PCT/IB2024/060428
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-10-24
Filing Date
2024-10-23
Publication Date
2025-05-01

AI Technical Summary

Technical Problem

Current treatments for neuromuscular degenerative disorders, such as ALS, are not curative and only modestly improve quality of life, with significant side effects, while also failing to address the formation and persistence of pathological protein aggregates and aberrant stress granules.

Method used

The use of an inhibitor of the METTL3-METTL14 complex or its pharmaceutically acceptable salts or solvates to treat neuromuscular degenerative pathologies, particularly those characterized by the accumulation of pathological protein aggregates or aberrant stress granules, either alone or in combination with other pharmaceutically active ingredients.

Benefits of technology

The inhibition of the METTL3-METTL14 complex reduces the formation of pathological stress granules, promotes their dissolution, delays disease progression, and relieves symptoms in neuromuscular degenerative disorders linked to mutations in the FUS gene.

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Abstract

The present invention relates to an inhibitor of the METTL3-METTL14 complex, or a pharmaceutically acceptable salt or solvate thereof, for use in the treatment of neuromuscular degenerative pathologies. These pathologies are preferably caused by or linked to mutations in the FUS gene and / or by the accumulation of pathological protein aggregates or aberrant stress granules. The inhibitor can be used alone or in combination with other pharmaceutically active ingredients. Additionally, the invention includes pharmaceutical compositions comprising the inhibitor and a pharmaceutically acceptable carrier or excipient, potentially combined with other agents known to treat or mitigate neuromuscular disorders.
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