- 专利标题: METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE
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申请号: US17935297申请日: 2022-09-26
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公开(公告)号: US20230285573A1公开(公告)日: 2023-09-14
- 发明人: Aseem ANSARI , Pratik SHAH
- 申请人: Design Therapeutics, Inc.
- 申请人地址: US CA Carlsbad
- 专利权人: Design Therapeutics, Inc.
- 当前专利权人: Design Therapeutics, Inc.
- 当前专利权人地址: US CA Carlsbad
- 主分类号: A61K47/59
- IPC分类号: A61K47/59 ; A61K31/454 ; A61K31/4545 ; A61K31/4704 ; A61K31/50 ; A61K31/502 ; A61K31/5025 ; A61K31/54 ; A61K48/00 ; A61K47/54 ; A61K47/60 ; A61K47/22 ; A61K47/56 ; A61K31/519 ; A61K31/517
摘要:
The present disclosure relates to compounds and methods for modulating the expression of bean (brain expressed, associated with NEDD4) and treating diseases and conditions in which bean plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence TGGAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence TGGAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.
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