JP7607884B2 - ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド - Google Patents

ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド Download PDF

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JP7607884B2
JP7607884B2 JP2023567797A JP2023567797A JP7607884B2 JP 7607884 B2 JP7607884 B2 JP 7607884B2 JP 2023567797 A JP2023567797 A JP 2023567797A JP 2023567797 A JP2023567797 A JP 2023567797A JP 7607884 B2 JP7607884 B2 JP 7607884B2
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bonds
nucleosides
antisense oligonucleotide
place
pharma
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JPWO2023112931A1 (https=
JPWO2023112931A5 (https=
Inventor
孝男 河野
俊明 小手
明咲 千葉
理 小野寺
泰介 加藤
祥子 廣川
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Nippon Shinyaku Co Ltd
Niigata University NUC
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Nippon Shinyaku Co Ltd
Niigata University NUC
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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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2023567797A 2021-12-13 2022-12-13 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド Active JP7607884B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024214710A JP2025038021A (ja) 2021-12-13 2024-12-09 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2021201756 2021-12-13
JP2021201756 2021-12-13
JP2022184853 2022-11-18
JP2022184853 2022-11-18
PCT/JP2022/045921 WO2023112931A1 (ja) 2021-12-13 2022-12-13 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド

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JP2024214710A Division JP2025038021A (ja) 2021-12-13 2024-12-09 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド

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JPWO2023112931A1 JPWO2023112931A1 (https=) 2023-06-22
JPWO2023112931A5 JPWO2023112931A5 (https=) 2024-07-01
JP7607884B2 true JP7607884B2 (ja) 2025-01-06

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JP2023567797A Active JP7607884B2 (ja) 2021-12-13 2022-12-13 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド
JP2024214710A Pending JP2025038021A (ja) 2021-12-13 2024-12-09 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド

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US (2) US20250051769A1 (https=)
EP (1) EP4450625A1 (https=)
JP (2) JP7607884B2 (https=)
TW (1) TW202340464A (https=)
WO (1) WO2023112931A1 (https=)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130198877A1 (en) 2010-08-05 2013-08-01 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
WO2019182037A1 (ja) 2018-03-20 2019-09-26 国立大学法人東京工業大学 毒性が低減されたアンチセンスオリゴヌクレオチド
WO2021153747A1 (ja) 2020-01-31 2021-08-05 株式会社三和化学研究所 Ａｔｎ１のアンチセンスオリゴヌクレオチド

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体，デバイス及び医薬組成物
JPWO2022211129A1 (https=) 2021-04-01 2022-10-06
WO2023164656A2 (en) 2022-02-25 2023-08-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atn1 expression
WO2023201281A2 (en) 2022-04-14 2023-10-19 University Of Massachusetts Oligonucleotides for atn1 modulation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130198877A1 (en) 2010-08-05 2013-08-01 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
WO2019182037A1 (ja) 2018-03-20 2019-09-26 国立大学法人東京工業大学 毒性が低減されたアンチセンスオリゴヌクレオチド
WO2021153747A1 (ja) 2020-01-31 2021-08-05 株式会社三和化学研究所 Ａｔｎ１のアンチセンスオリゴヌクレオチド

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
EVERS, M.M., et al.，Targeting several CAG expansion diseases by a single antisense oligonucleotide.，PLoS One，2011年，Vol.6, No.9, e24308，pp.1-11
HU, J., et al.，Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.，Biochemistry，2014年，Vol.53, No.28，pp.4510-4518

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EP4450625A1 (en) 2024-10-23
TW202340464A (zh) 2023-10-16
US12291710B2 (en) 2025-05-06
US20240318181A1 (en) 2024-09-26
WO2023112931A1 (ja) 2023-06-22
JP2025038021A (ja) 2025-03-18
JPWO2023112931A1 (https=) 2023-06-22
US20250051769A1 (en) 2025-02-13

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