Heterocyclic and heteroaryl compounds for treating Huntington's disease

Substituted bicyclic heterocyclic and heteroaryl compounds are developed to treat Huntington's disease, addressing the lack of small-molecule therapies by effectively targeting the disease's underlying cause and offering potential combination therapy benefits.

JP2025522298APending Publication Date: 2025-07-15PTC THERAPEUTICS INC
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Patent Information

Application Number
JP2024569340
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2022-06-15
Filing Date
2023-06-13
Publication Date
2025-07-15

AI Technical Summary

Technical Problem

There is currently no small-molecule therapy available to target the underlying cause of Huntington's disease, a progressive neurodegenerative disorder, and there is a high unmet need for drugs that can treat or ameliorate the symptoms.

Method used

Development of substituted bicyclic heterocyclic and heteroaryl compounds, represented by formula (I), which can be administered to subjects to treat or ameliorate Huntington's disease, potentially in combination with other agents, and exist in various forms such as salts, hydrates, and stereoisomers.

Benefits of technology

The compounds effectively target the underlying cause of Huntington's disease, providing therapeutic benefits and can be used in combination therapies to enhance treatment efficacy.

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Abstract

This specification relates to compounds, their forms and pharmaceutical compositions, and methods of using such compounds, their forms or compositions for treating or ameliorating Huntington's disease. JPEG2025522298000094.jpg3647In particular, this specification relates to substituted bicyclic heterocyclic and heteroaryl compounds of formula (I), their forms and pharmaceutical compositions, and methods of using such compounds, their forms or compositions for treating or ameliorating Huntington's disease.
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