Title:
ADENO-ASSOCIATED VIRUS FACTOR VIII VECTORS
Document Type and Number:
Japanese Patent JP2023133597
Kind Code:
A
Abstract:
To provide novel AAV vector encoding functional factor VIII protein useful in gene therapy for treating hemophilia A.SOLUTION: The present invention provides AAV vectors (referred to herein as "AAV FVIII vectors") that encode functionally active FVIII. The genome encoding the functionally active FVIII is preferably 7.0 kb in length or less, more preferably 6.5 kb in length or less, even more preferably 6.0 kb in length or less, even more preferably 5.5 kb in length or less, and even more preferably 5.0 kb in length or less, and has enhanced promoter function. The present invention also relates to methods for making the AAV FVIII vector described herein and related therapeutic uses.SELECTED DRAWING: None
Inventors:
PETER CAMERON COLOSI
AMIT NATHWANI
JENNY MCINTOSH
EDWARD TUDDENHAM
ANDREW DAVIDOFF
AMIT NATHWANI
JENNY MCINTOSH
EDWARD TUDDENHAM
ANDREW DAVIDOFF
Application Number:
JP2023127698A
Publication Date:
September 22, 2023
Filing Date:
August 04, 2023
Export Citation:
Assignee:
BIOMARIN PHARM INC
UNIV LONDON
ST JUDE CHILDRENS RES HOSPITAL
UNIV LONDON
ST JUDE CHILDRENS RES HOSPITAL
International Classes:
C12N15/864; A61K35/76; A61K38/37; A61K48/00; A61P7/04; A61P43/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N7/01
Attorney, Agent or Firm:
Shusaku Yamamoto
Natsuki Morishita
Natsuki Morishita
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