Nucleic acid complex and pharmaceutical composition containing same

Patent metadata

Field	Value
Publication number	US-2023151360-A1
Application number	US-202117791343-A
Country	US
Kind code	A1
Filing date	Jan 28, 2021
Priority date	Jan 30, 2020
Publication date	May 18, 2023
Grant date	—

Abstract

Disclosed is a nucleic acid complex or a pharmaceutically acceptable salt thereof; the nucleic acid complex represented by the formula (I), wherein X is CH2 or O; Y is a sugar ligand having mannose or GalNAc; n is an integer of 1 to 8; and Z is a group comprising an oligonucleotide.

First claim

1 . A nucleic acid complex represented by the formula (I):

or the formula (II):

wherein X is CH 2 or O;
Y is a sugar ligand having mannose or GalNAc;
n is an integer of 1 to 8; and
Z is a group comprising an oligonucleotide,
or a pharmaceutically acceptable salt thereof.

2 . (canceled)

3 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (III):

wherein Z is a group comprising an oligonucleotide.

4 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (IV):

wherein Z is a group comprising an oligonucleotide.

5 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (V):

wherein Z is a group comprising an oligonucleotide.

6 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (VI):

wherein Z is a group comprising an oligonucleotide.

7 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (VII):

wherein Z is a group comprising an oligonucleotide.

8 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (VIII):

wherein Z is a group comprising an oligonucleotide.

9 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (IX):

wherein Z is a group comprising an oligonucleotide.

10 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (X):

wherein Z is a group comprising an oligonucleotide.

11 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (XI):

wherein Z is a group comprising an oligonucleotide.

12 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (XII):

wherein Z is a group comprising an oligonucleotide.

13 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (XIII):

wherein Z is a group comprising an oligonucleotide.

14 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the nucleic acid complex is represented by the formula (XIV):

wherein Z is a group comprising an oligonucleotide.

15 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the oligonucleotide is single-stranded.

16 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 15 , wherein the oligonucleotide is bound via a 3′ end.

17 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 15 , wherein the oligonucleotide is bound via a 5′ end.

18 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 , wherein the oligonucleotide is double-stranded.

19 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 18 , wherein the oligonucleotide is bound via a 3′ end of one strand.

20 . The nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 18 , wherein the oligonucleotide is bound via a 5′ end of one strand.

21 . A pharmaceutical composition comprising the nucleic acid complex or pharmaceutically acceptable salt thereof according to claim 1 .

22 .- 23 . (canceled)

Assignees

• Eisai R&D Man Co Ltd

Inventors

• Suzuki Yuta
• Yamazaki Kazuto
• KUBARA Kenji
• Tamura Tomohiko
• Kurotaki Daisuke

Classifications

• C12N15/111 Primary

  General methods applicable to biologically active non-coding nucleic acids · CPC title

• C12N2310/315

  Phosphorothioates · CPC title

• C12N15/113 Primary

  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

• C12N2310/14

  interfering nucleic acids [NA] · CPC title

• C12N2310/351

  Conjugate · CPC title