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Title:
LIGAND-BOUND NUCLEIC ACID COMPLEX
Document Type and Number:
WIPO Patent Application WO/2022/255273
Kind Code:
A1
Abstract:
Provided is an S1P ligand-bound nucleic acid complex that is delivered to a desired organ to regulate the expression or editing of a target gene, a transcript thereof or a translation product thereof. This S1P ligand-bound nucleic acid complex, which has a structure wherein an S1P ligand is bound to a nucleic acid, said nucleic acid containing an antisense strand containing an oligonucleotide that has a nucleic acid base sequence complementary to a target transcript and consists of 12-30 nucleotides, is delivered to an organ and regulates the expression or editing of a target gene, a transcript thereof or a translation product thereof, which makes the complex useful in treating a disease caused by the gene. Further, this ligand-bound nucleic acid complex can be delivered to organs, tissues and cells in which S1P receptors are expressed, for example, skeletal muscle, cardiac muscle, liver, kidney, lung, mammary gland, fat, podocytes, lymphocytes and vascular endothelial cells, which makes the complex useful in treating diseases in these organs.

Inventors:
TAKAGI KOH (JP)
KIZAWA HIDEKI (JP)
OKAMOTO HIROSHI (JP)
KANEKO KEIKO (JP)
HUSE NOBUYUKI (JP)
OCHI TAKASHI (JP)
Application Number:
PCT/JP2022/021836
Publication Date:
December 08, 2022
Filing Date:
May 27, 2022
Export Citation:
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Assignee:
RENA THERAPEUTICS INC (JP)
International Classes:
A61K47/54; A61K31/7088; A61K31/7105; A61K31/713; A61P43/00; C12N15/113; C12N15/115
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Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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