Nucleoside derivative and use thereof

Patent metadata

Field	Value
Publication number	US-12398170-B2
Application number	US-202017771089-A
Country	US
Kind code	B2
Filing date	Oct 28, 2020
Priority date	Oct 28, 2019
Publication date	Aug 26, 2025
Grant date	Aug 26, 2025

Abstract

Provided is a nucleoside derivative represented by the following formula (1):

or a salt thereof,
wherein R 1 represents an alkoxy group, a hydrogen atom or a halogen atom; R 2 and R 4 , which may be the same as or different from each other, each represents a hydrogen atom, a protective group for a hydroxyl group, a phosphate group, a protected phosphate group, or —P(═O) n R 5 R 6 in which n represents 0 or 1, R 5 and R 6 , which may be the same as or different from each other, each represents a hydrogen atom, a hydroxyl group, a protected hydroxyl group, a mercapto group, a protected mercapto group, an alkoxy group, a cyanoalkoxy group, an amino group, or a substituted amino group, provided that when n is 1, both R 5 and R 6 cannot be the hydrogen atom at the same time; R 3 represents —(CH 2 ) m NHR 7 in which m represents an integer of 1 to 6, R 7 represents a hydrogen atom, an alkyl group, an alkenyl group or a protective group for an amino group; and B represents a purin-9-yl group, a 2-oxo-pyrimidin-1-yl group, a substituted purin-9-yl group, or a substituted 2-oxo-pyrimidin-1-yl group.

First claim

The invention claimed is:

1. A nucleoside derivative represented by the following formula (1):

or a salt thereof,
wherein R 1 represents a methoxy group; R 2 and R 4 , which may be the same as or different from each other, each represents a hydrogen atom, a protective group for a hydroxyl group, a phosphate group, a protected phosphate group, or —P (=O) n R 5 R 6 in which n represents 0 or 1, R 5 and R 6 , which may be the same as or different from each other, each represents a hydrogen atom, a hydroxyl group, a protected hydroxyl group, a mercapto group, a protected mercapto group, an alkoxy group, a cyanoalkoxy group, an amino group, or a substituted amino group, provided that when n is 1, both R 5 and R 6 cannot be the hydrogen atom at the same time; R 3 represents —(CH 2 ) m NHR 7 in which m represents 2, R 7 represents a hydrogen atom, an alkyl group, an alkenyl group or a protective group for an amino group; and B represents a purin-9-yl group, a 2-oxo-pyrimidin-1-yl group, a substituted purin-9-yl group, or a substituted 2-oxo-pyrimidin-1-yl group.

2. The nucleoside derivative or the salt thereof according to claim 1 , wherein R 7 represents a hydrogen atom.

3. An oligonucleotide derivative comprising at least one partial structure represented by the following formula (2):

or a salt thereof,
wherein, R 1 represents a methoxy group; R 3 represents —(CH 2 ) m NHR 7 in which m represents 2, and R 7 represents a hydrogen atom, an alkyl group, an alkenyl group, or a protective group for an amino group; and B represents a purin-9-yl group, a 2-oxo-pyrimidin-1-yl group, a substituted purin-9-yl group, or a substituted 2-oxo-pyrimidin-1-yl group.

4. The oligonucleoside derivative or the salt thereof according to claim 3 , wherein R 7 represents a hydrogen atom.

5. An siRNA comprising the oligonucleotide derivative or the salt thereof according to claim 3 as an active ingredient.

Assignees

• National Univ Corporation Tokai National Higher Education And Research System
• Yamasa Corp

Inventors

• Ueno Yoshihito

Classifications

• C12N2310/14

  interfering nucleic acids [NA] · CPC title

• C12N15/113

  Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title

• C07H21/02

  with ribosyl as saccharide radical · CPC title

• C12N2310/3527

  Other alkyl chain · CPC title

• C12N2310/32

  of the sugar · CPC title