Title:
IMPROVED HUMAN COAGULATION FACTOR VIII GENE EXPRESSION CASSETTE AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/060463
Kind Code:
A1
Abstract:
The present invention relates to an optimized polynucleotide sequence encoding a human coagulation factor VIII (FVIII) polypeptide. The present disclosure also relates to an FVIII gene expression cassette, a recombinant AAV vector, and a pharmaceutical composition comprising the optimized polynucleotide sequence, and use thereof in the treatment of hemophilia A or acquired factor VIII deficiency. The optimized polynucleotide sequence and the FVIII gene expression cassette of the present invention have an improved FVIII expression level.
Inventors:
HU HANYANG (CN)
LI DANDAN (CN)
XU YUE (CN)
CHEN CHEN (CN)
WANG TIANYI (CN)
YUAN LONGHUI (CN)
WANG XINTAO (CN)
DU ZENGMIN (CN)
JIANG WEI (CN)
WU XIA (CN)
ZHENG JING (CN)
XIAO XIAO (CN)
LI DANDAN (CN)
XU YUE (CN)
CHEN CHEN (CN)
WANG TIANYI (CN)
YUAN LONGHUI (CN)
WANG XINTAO (CN)
DU ZENGMIN (CN)
JIANG WEI (CN)
WU XIA (CN)
ZHENG JING (CN)
XIAO XIAO (CN)
Application Number:
PCT/CN2022/143456
Publication Date:
March 28, 2024
Filing Date:
December 29, 2022
Export Citation:
Assignee:
SHANGHAI MYGT BIOPHARMACEUTICAL LLC (CN)
International Classes:
C12N15/12; A61K38/37; A61K48/00; A61P7/04; C12N5/10; C12N15/864
Foreign References:
| CN107531774A | 2018-01-02 | |||
| CN108884145A | 2018-11-23 | |||
| CN112538501A | 2021-03-23 |
Other References:
LIRON ELKOUBY ET AL.: "Preclinical Assessment of an Optimized AAV-FVIII Vector in Mice and Non-human Primates for the Treatment of Hemophilia A", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 24, 10 March 2022 (2022-03-10), pages 20 - 29, XP093069094, DOI: 10.1016/j.omtm.2021.11.005
LI-JUN ZHONG, JIANG WEI, WANG FENG-SHAN, ZANG HENG-CHANG, : "Research Progress on Human Coagulation Factor Ⅷ Chinese Full Tex", FOOD AND DRUG, vol. 16, no. 3, 20 May 2014 (2014-05-20), pages 212 - 214, XP093149605
LI-JUN ZHONG, JIANG WEI, WANG FENG-SHAN, ZANG HENG-CHANG, : "Research Progress on Human Coagulation Factor Ⅷ Chinese Full Tex", FOOD AND DRUG, vol. 16, no. 3, 20 May 2014 (2014-05-20), pages 212 - 214, XP093149605
Attorney, Agent or Firm:
WATSON & BAND INTELLECTUAL PROPERTY AGENT LTD. (CN)
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