WO2025088552 - NUCLEIC ACID CONSTRUCTS AND USES THEREOF
National phase entry:
Publication Number
WO/2025/088552
Publication Date
01.05.2025
International Application No.
PCT/IB2024/060500
International Filing Date
25.10.2024
Title **
[English]
NUCLEIC ACID CONSTRUCTS AND USES THEREOF
[French]
CONSTRUCTIONS D'ACIDES NUCLÉIQUES ET LEURS UTILISATIONS
Applicants **
KANGLIN BIOTECH (HANGZHOU) CO., LTD.
Inventors
WU, Haoquan
DANG, Ying
SU, Lingling
YE, Qing
TAO, Jianjun
HU, Xiaodong
JIANG, Chao
Priority Data
18/383,596
25.10.2023
US
Application details
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Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 3180 | |
| EPO | Filing, Examination, Granting | 18575 | |
| Japan | Filing, Examination, Granting | 2735 | |
| South Korea | Filing, Examination, Granting | 3339 | |
| USA | Filing, Examination, Granting | 8990 |
Total:
36,819
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 gene therapy vectors used for efficiently transducing cells to express a human β-globin gene. Specifically disclosed is an expression vector which comprises: an expression cassette for a β-globin gene, which comprises exons and introns of human β-globin gene, as well as cis-acting elements including one or more of WPRE, SV40 polyadenylation signal and/or SV40 ori. The disclosed expression vectors have significantly enhanced viral vector packaging efficiency in viral vector packaging cell lines, which leads to effective integration of lentiviral vectors and high expression level of β-globin gene in target cells. Also disclosed are pharmaceutical compositions and therapeutic methods utilizing such expression vectors.[French]
Sont divulgués des vecteurs de thérapie génique utilisés pour transduire efficacement des cellules pour exprimer un gène de β-globine humaine. Est spécifiquement divulgué un vecteur d'expression qui consiste en : une cassette d'expression pour un gène de β-globine, qui consiste en des exons et des introns du gène de la β-globine humaine, ainsi que des éléments à action cis consistant en un ou plusieurs des signaux de polyadénylation WPRE, SV40 et/ou SV40 ori. Les vecteurs d'expression divulgués présentent une efficacité d'encapsulation de vecteur viral considérablement améliorée dans des lignées cellulaires d'encapsidation de vecteur viral, ce qui conduit à une intégration efficace de vecteurs lentiviraux et à un niveau d'expression élevé de gène de β-globine dans des cellules cibles. Sont également divulguées des compositions pharmaceutiques et des méthodes thérapeutiques utilisant de tels vecteurs d'expression.