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Title:
SEQUENCE OF ACTIVATED PROTEIN C
Document Type and Number:
WIPO Patent Application WO/2023/171719
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a breakthrough activated protein C that can cure protein C deficiency, a pharmaceutical composition containing the same, etc. The present invention provides a polypeptide, which contains an amino acid sequence represented by formula (I): A1-A2-A3 (wherein: A1 represents an amino acid sequence containing the amino acid sequence of the light chain of protein C or a homologue thereof; A2 represents an amino acid sequence constituting a self-cleavage site; and A3 represents an amino acid sequence containing the amino acid sequence of the heavy chain of protein C or a homologue thereof) or a partial polypeptide thereof, wherein the dimeric protein consisting of the N-terminal fragment and the C-terminal fragment of the cleavage site of A2 or a partial protein thereof has protein C activity. This polypeptide or a partial polypeptide thereof enables: 1) the production of the activated protein C in the form of a recombinant preparation; and 2) the use thereof in effective gene therapy for protein C deficiency.

Inventors:
OHMORI TSUKASA (JP)
TOGASHI TOMOKI (JP)
HAYAKAWA MORISADA (JP)
KAMOSHITA NOBUHIKO (JP)
KASHIWAKURA YUJI (JP)
HIRAMOTO TAKAFUMI (JP)
BAATARTSOGT NEMEKHBAYAR (JP)
Application Number:
PCT/JP2023/008880
Publication Date:
September 14, 2023
Filing Date:
March 08, 2023
Export Citation:
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Assignee:
UNIV JICHI MEDICAL (JP)
International Classes:
C12N15/57; A61K35/76; A61K38/36; A61K48/00; A61P7/02; A61P43/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N9/64; C12N15/864
Domestic Patent References:
WO2001070763A12001-09-27
WO2000066754A12000-11-09
Foreign References:
JP2000501928A2000-02-22
JPH022372A1990-01-08
JP2016518840A2016-06-30
Attorney, Agent or Firm:
KAMADA, Mitsunori et al. (JP)
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