WO2023210233A1 - Polynucléotide lié à un sucre - Google Patents

Polynucléotide lié à un sucre Download PDF

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Publication number
WO2023210233A1
WO2023210233A1 PCT/JP2023/012164 JP2023012164W WO2023210233A1 WO 2023210233 A1 WO2023210233 A1 WO 2023210233A1 JP 2023012164 W JP2023012164 W JP 2023012164W WO 2023210233 A1 WO2023210233 A1 WO 2023210233A1
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WO
WIPO (PCT)
Prior art keywords
compound
group
salt
polynucleotide
solvate
Prior art date
Application number
PCT/JP2023/012164
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English (en)
Japanese (ja)
Inventor
哲也 兒玉
義大 百相
永士 三森
春彦 鎌田
泰亮 中山
Original Assignee
国立大学法人東海国立大学機構
国立研究開発法人医薬基盤・健康・栄養研究所
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 国立大学法人東海国立大学機構, 国立研究開発法人医薬基盤・健康・栄養研究所 filed Critical 国立大学法人東海国立大学機構
Publication of WO2023210233A1 publication Critical patent/WO2023210233A1/fr

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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

Abstract

La présente invention aborde le problème de la fourniture d'une technique permettant d'exercer de manière efficace l'activité d'un polynucléotide dans le cerveau. La solution selon l'invention porte sur un composé représenté par la formule générale (1) [dans la formule, Ms représente un groupe monovalent obtenu par élimination d'un atome ou d'un groupe d'un monosaccharide. Lk représente un groupe divalent qui est un lieur. Pn représente un groupe monovalent obtenu par élimination d'un atome ou d'un groupe d'un polynucléotide.], ou un sel ou des solvates de celui-ci.
PCT/JP2023/012164 2022-04-27 2023-03-27 Polynucléotide lié à un sucre WO2023210233A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2022073180 2022-04-27
JP2022-073180 2022-04-27

Publications (1)

Publication Number Publication Date
WO2023210233A1 true WO2023210233A1 (fr) 2023-11-02

Family

ID=88518690

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/JP2023/012164 WO2023210233A1 (fr) 2022-04-27 2023-03-27 Polynucléotide lié à un sucre

Country Status (1)

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WO (1) WO2023210233A1 (fr)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015075942A1 (fr) * 2013-11-22 2015-05-28 国立大学法人 東京大学 Vecteur d'apport de médicament et conjugué, composition le contenant, et son procédé d'administration

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015075942A1 (fr) * 2013-11-22 2015-05-28 国立大学法人 東京大学 Vecteur d'apport de médicament et conjugué, composition le contenant, et son procédé d'administration

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Practical Application of Five Types of Reagents Contributing to Nucleic Acid Therapeutics (RNA) Life Prolongation and Anticancer Activity - Manufacture/Sale Agreement for Nucleic Acid Therapeutic Study Reagent Executed with Kanto Kagaku - Streamlining R&D with Safe and Easy Post-Modification of RNA ", PRESS RELEASE OF TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH SYSTEM, GIFU UNIVERSITY, 24 May 2016 (2016-05-24), XP093105533 *
KUWAHARA HIROYA, TAKANORI YOKOTA: "Efficient DDS that crosses the blood-brain barrier in response to changes in blood glucose concentration", DRUG DELIVERY SYSTEM, vol. 34, no. 5, 1 January 2019 (2019-01-01), pages 352 - 359, XP093105528, DOI: 10.2745/dds.34.352 *
LIAO WENQING, AKAHIRA SAKIKO, HARA RINTARO IWATA, WADA TAKESHI, KUSAMORI KOSUKE, TAKAKURA YOSHINOBU, NISHIKAWA MAKIYA: "Enhanced Immunostimulatory Activity of CpG Oligodeoxynucleotide by the Combination of Mannose Modification and Incorporation into Nanostructured DNA", BIOLOGICAL & PHARMACEUTICAL BULLETIN, PHARMACEUTICAL SOCIETY OF JAPAN, TOKYO., JP, vol. 43, no. 8, 1 August 2020 (2020-08-01), JP , pages 1188 - 1195, XP093105521, ISSN: 0918-6158, DOI: 10.1248/bpb.b20-00052 *
NAGATA TETSUYA; DWYER CHRISSA A.; YOSHIDA-TANAKA KIE; IHARA KENSUKE; OHYAGI MASAKI; KABURAGI HIDETOSHI; MIYATA HARUKA; EBIHARA SAT: "Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 39, no. 12, 12 August 2021 (2021-08-12), New York, pages 1529 - 1536, XP037639828, ISSN: 1087-0156, DOI: 10.1038/s41587-021-00972-x *
NISHIKAWA MAKIYA, YUKITAKE YOSHIOKA, MAKOTO NAGAOKA, KOSUKE KUSAMORI: "Development of Oligonucleotide Therapeutics: Tissue Distribution and Drug Delivery System", DDS. DRUG DELIVERY SYSTEM, vol. 36, no. 1, 25 January 2021 (2021-01-25), pages 40 - 50, XP093006747, DOI: 10.2745/dds.36.40 *
TETSUYA KODAMA, YOSHIHIRO HYAKUSO, EIJI MIMORI, SHUICHI HIROAKI, RISA HYAKUSO, TATSUYA CHIKADA: "1P-19 Usefulness and cautionary points of post-synthetic modification of oligonucleotides by click", PROCEEDINGS OF THE 6TH ANNUAL MEETING OF THE NUCLEIC ACIDS THERAPEUTICS SOCIETY OF JAPAN; JUNE 27TH TO 29TH, 2021, NUCLEIC ACIDS THERAPEUTICS SOCIETY OF JAPAN, JP, vol. 6, 1 January 2021 (2021-01-01) - 29 June 2021 (2021-06-29), JP, pages 101, XP009550138 *

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