WO2025004001 - HTT REPRESSORS AND USES THEREOF
National phase entry is expected:
Publication Number
WO/2025/004001
Publication Date
02.01.2025
International Application No.
PCT/IB2024/056361
International Filing Date
28.06.2024
Title **
[English]
HTT REPRESSORS AND USES THEREOF
[French]
RÉPRESSEURS DE HTT ET LEURS UTILISATIONS
Applicants **
TAKEDA PHARMACEUTICAL COMPANY LIMITED
Inventors
MOORE, Simon
PROETZEL, Gabriele
DUONG, Khanh
IWATA, Hidehisa
Priority Data
63/511,437
30.06.2023
US
Application details
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International Searching Authority |
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| Recordal of a Change of the Applicant's Name/Address | * |
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| 译文 |
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* The data is based on automatic recognition. Please verify and amend if necessary.
** IP-Coster compiles data from publicly available sources. If this data includes your personal information, you can contact us to request its removal.
Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 3348 | |
| EPO | Filing, Examination, Granting | 23481 | |
| Japan | Filing, Examination, Granting | 3085 | |
| South Korea | Filing, Examination, Granting | 4143 | |
| USA | Filing, Examination, Granting | 14790 |
Total:
48,847
The term for entry into the National Phase has expired. This quotation is for informational purposes only
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Abstract[English]
Disclosed herein are improved methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. Among other things, provided herein is a gene therapy construct encoding a non-naturally occurring codon-optimized transcription factor (ZFP-TF) comprising a zinc-finger protein (ZFP) sequence and a sequence encoding a transcriptional repression domain, wherein the ZFP-TF expression is driven by a phosphoglycerate kinase 1 (PGK), ubiquitin C (UBC), an EFS, or an EF1alpha promoter.[French]
Sont divulguées des méthodes et des compositions améliorées pour le diagnostic, la prévention et/ou le traitement de la maladie de Huntington. Entre autres, l'invention concerne une construction de thérapie génique codant pour un facteur de transcription à codons optimisés d'origine non naturelle (ZFP-TF) comprenant une séquence de protéine à doigt de zinc (ZFP) et une séquence codant pour un domaine de répression transcriptionnelle, l'expression de ZFP-TF étant entraînée par une phosphoglycérate kinase 1 (PGK), l'ubiquitine C (UBC), un EFS, ou un promoteur EF1alpha.