STEM CELLS EDITED WITH FE-FVIII MUTANT GENE, ENDOTHELIAL CELLS DIFFERENTIATED THEREFROM, AND PHARMACEUTICAL COMPOSITION CONTAINING SAME FOR PREVENTION OR TREATMENT OF HEMOPHILIA

Title:

STEM CELLS EDITED WITH FE-FVIII MUTANT GENE, ENDOTHELIAL CELLS DIFFERENTIATED THEREFROM, AND PHARMACEUTICAL COMPOSITION CONTAINING SAME FOR PREVENTION OR TREATMENT OF HEMOPHILIA

Document Type and Number:

WIPO Patent Application WO/2022/211604

Kind Code:

A1

Abstract:

The present invention relates to stem cells edited with a FE-FVIII mutant gene, endothelial cells differentiated therefrom, and a pharmaceutical composition containing same for prevention or treatment of hemophilia. The composition, the pluripotent stem cells, and the endothelial cells differentiated therefrom according to the present invention have an excellent activity of recovering the function of FVIII and thus can be advantageously used as a therapeutic agent for hemophilia.

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Inventors:

KIM DONG-WOOK (KR)
PARK CHUL-YONG (KR)
CHOI SANG-HWI (KR)
KIM DO-HUN (KR)

Application Number:

PCT/KR2022/004816

Publication Date:

October 06, 2022

Filing Date:

April 04, 2022

Export Citation:

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Assignee:

UNIV YONSEI IACF (KR)

International Classes:

C12N5/074; A61K35/44; A61K48/00; A61P7/04; C12N5/071; C12N9/22; C12N15/10

Foreign References:

KR20200011817A	2020-02-04
KR20180070699A	2018-06-26
US20200384125A1	2020-12-10

Other References:

ORLOVA N. A., KOVNIR S. V., VOROBIEV I. I., GABIBOV A. G., VOROBIEV A. I.: "Blood Clotting Factor VIII: From Evolution to Therapy", ACTA NATURAE, PARK MEDIA, RU, vol. 5, no. 2, 1 January 2013 (2013-01-01), RU , pages 19 - 39, XP055971966, ISSN: 2075-8251, DOI: 10.32607/20758251-2013-5-2-19-39
FANTACINI DAIANNE MACIELY CARVALHO, FONTES APARECIDA MARIA, DE ABREU NETO MÁRIO SOARES, COVAS DIMAS TADEU, PICANÇO-CASTRO VIRGÍNIA: "The F309S mutation increases factor VIII secretion in human cell line", REVISTA BRASILEIRA DE HEMATOLOGIA E HEMOTERAPIA, SOCIEDADE BRASILEIRA DE HEMATOLOGIA E HEMOTERAPIA ;, vol. 38, no. 2, 20 April 2016 (2016-04-20), pages 135 - 140, XP055971969, ISSN: 1516-8484, DOI: 10.1016/j.bjhh.2016.04.002

Attorney, Agent or Firm:

KONG, Byung Wook (KR)

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