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Title:
NUCLEIC ACID HAVING CARRIER PEPTIDE LINKED THERETO
Document Type and Number:
WIPO Patent Application WO/2023/171820
Kind Code:
A1
Abstract:
In the present description, there are provided; a nucleic acid having a carrier peptide linked thereto, or a pharmaceutically acceptable salt of the nucleic acid, or a hydrate of the nucleic acid or the pharmaceutically acceptable salt; and others. The nucleic acid having a carrier peptide linked thereto or the pharmaceutically acceptable salt of the nucleic acid or the hydrate of the nucleic acid or the pharmaceutically acceptable salt comprises: a nucleic acid capable of regulating the splicing of pre-mRNA or inhibiting the function of targeted RNA or a target region in the RNA or a pharmaceutically acceptable salt of the nucleic acid or a hydrate of the nucleic acid or the pharmaceutically acceptable salt; and a carrier peptide that is linked to the nucleic acid, the pharmaceutically acceptable salt of the nucleic acid or the hydrate of the nucleic acid or the pharmaceutically acceptable salt and comprises the amino acid sequence represented by the formula KKRTLRKSNRKKR (SEQ ID NO:1) or an amino acid sequence having such a structure that one or two amino acid residues in SEQ ID NO:1 are added, deleted or substituted (provided that an amino acid sequence in which both of amino acid residues located at position-8 and position-9 from the N-terminal of SEQ ID NO:1 are substituted is excluded). In the nucleic acid having a carrier peptide linked thereto, or a pharmaceutically acceptable salt of the nucleic acid, or a hydrate of the nucleic acid or the pharmaceutically acceptable salt, the nucleic acid is selected from the group consisting of an antisense oligomer, siRNA and shRNA.

Inventors:
SUGIYAMA HIROYUKI (JP)
NAKAMURA TAKAHIRO (JP)
NOGATA MASAKI (JP)
KOMIYAMA KEINA (JP)
KAWANO TAKAO (JP)
FUJIWARA MIKIYA (JP)
BAILEYKOBAYASHI NAHOKO (JP)
YOSHIDA TETSUHIKO (JP)
Application Number:
PCT/JP2023/009470
Publication Date:
September 14, 2023
Filing Date:
March 10, 2023
Export Citation:
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Assignee:
NIPPON SHINYAKU CO LTD (JP)
International Classes:
C12N15/113; A61K9/127; A61K31/7105; A61K31/713; A61K31/80; A61K47/64; A61K48/00; A61P21/04; C07K7/08; C12N15/87
Domestic Patent References:
WO2011013700A12011-02-03
WO2013100190A12013-07-04
WO2012029986A12012-03-08
WO2021125311A12021-06-24
Foreign References:
JP2022014707A2022-01-20
Other References:
GOYAL PANKAJ , PANDEY DHARMENDRA, SIESS WOLFGANG: "Phosphorylation-dependent regulation of unique nuclear and nucleolar localization signals of LIM kinase 2 in endothelial cells.", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 281, no. 35, 1 September 2006 (2006-09-01), US , pages 25223 - 25230, XP002622536, ISSN: 0021-9258, DOI: 10.1074/JBC.M603399200
Attorney, Agent or Firm:
KOBAYASHI Hiroshi et al. (JP)
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