JPWO2023112931A1 - - Google Patents
Info
- Publication number
- JPWO2023112931A1 JPWO2023112931A1 JP2023567797A JP2023567797A JPWO2023112931A1 JP WO2023112931 A1 JPWO2023112931 A1 JP WO2023112931A1 JP 2023567797 A JP2023567797 A JP 2023567797A JP 2023567797 A JP2023567797 A JP 2023567797A JP WO2023112931 A1 JPWO2023112931 A1 JP WO2023112931A1
- Authority
- JP
- Japan
- Legal status (The legal status 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 status listed.)
- Granted
Links
Classifications
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- 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
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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
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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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
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- 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
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- 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
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- 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
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- 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
Landscapes
- 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)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (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)
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JPWO2023112931A1 true JPWO2023112931A1 (https=) | 2023-06-22 |
| JPWO2023112931A5 JPWO2023112931A5 (https=) | 2024-07-01 |
| JP7607884B2 JP7607884B2 (ja) | 2025-01-06 |
Family
ID=86774345
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US20250051769A1 (https=) |
| EP (1) | EP4450625A1 (https=) |
| JP (2) | JP7607884B2 (https=) |
| TW (1) | TW202340464A (https=) |
| WO (1) | WO2023112931A1 (https=) |
Citations (3)
| 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)
| 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 |
-
2022
- 2022-12-13 WO PCT/JP2022/045921 patent/WO2023112931A1/ja not_active Ceased
- 2022-12-13 EP EP22907455.4A patent/EP4450625A1/en active Pending
- 2022-12-13 JP JP2023567797A patent/JP7607884B2/ja active Active
- 2022-12-13 US US18/719,037 patent/US20250051769A1/en active Pending
- 2022-12-13 TW TW111147856A patent/TW202340464A/zh unknown
-
2024
- 2024-06-12 US US18/741,110 patent/US12291710B2/en active Active
- 2024-12-09 JP JP2024214710A patent/JP2025038021A/ja active Pending
Patent Citations (3)
| 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)
| Title |
|---|
| EVERS, M.M., ET AL.: "Targeting several CAG expansion diseases by a single antisense oligonucleotide.", PLOS ONE, vol. Vol.6, No.9, e24308, JPN6023007340, 2011, pages 1 - 11, ISSN: 0005371136 * |
| HU, J., ET AL.: "Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.", BIOCHEMISTRY, vol. 53, no. 28, JPN6023007339, 2014, pages 4510 - 4518, ISSN: 0005371137 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2025038021A (ja) | 2025-03-18 |
| WO2023112931A1 (ja) | 2023-06-22 |
| JP7607884B2 (ja) | 2025-01-06 |
| EP4450625A1 (en) | 2024-10-23 |
| US20240318181A1 (en) | 2024-09-26 |
| TW202340464A (zh) | 2023-10-16 |
| US12291710B2 (en) | 2025-05-06 |
| US20250051769A1 (en) | 2025-02-13 |
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