JPWO2022097727A1 - - Google Patents
Info
- Publication number
- JPWO2022097727A1 JPWO2022097727A1 JP2022560831A JP2022560831A JPWO2022097727A1 JP WO2022097727 A1 JPWO2022097727 A1 JP WO2022097727A1 JP 2022560831 A JP2022560831 A JP 2022560831A JP 2022560831 A JP2022560831 A JP 2022560831A JP WO2022097727 A1 JPWO2022097727 A1 JP WO2022097727A1
- 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.)
- Pending
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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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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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- 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
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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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020185944 | 2020-11-06 | ||
| PCT/JP2021/040853 WO2022097727A1 (ja) | 2020-11-06 | 2021-11-05 | Ｒｐｓ２５遺伝子の発現及び／又は機能調節剤 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JPWO2022097727A1 true JPWO2022097727A1 (https=) | 2022-05-12 |
| JPWO2022097727A5 JPWO2022097727A5 (https=) | 2023-11-10 |
Family
ID=81458361
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022560831A Pending JPWO2022097727A1 (https=) | 2020-11-06 | 2021-11-05 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20240018516A1 (https=) |
| EP (1) | EP4252846A4 (https=) |
| JP (1) | JPWO2022097727A1 (https=) |
| CN (1) | CN116507727A (https=) |
| TW (1) | TWI910261B (https=) |
| WO (1) | WO2022097727A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPWO2024071099A1 (https=) * | 2022-09-29 | 2024-04-04 | ||
| TW202511485A (zh) | 2023-06-16 | 2025-03-16 | 日商住友製藥股份有限公司 | 調節ｒｐｓ２５基因之表現及／或其功能的反義寡核苷酸 |
| WO2025047953A1 (ja) * | 2023-08-30 | 2025-03-06 | 株式会社Stratoimmune | ＲａｓＧＲＰ４のアンチセンスオリゴヌクレオチド |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005536230A (ja) * | 2002-04-24 | 2005-12-02 | エクスプレッション ダイアグノスティクス， インコーポレイテッド | 移植拒絶を診断およびモニタリングするための方法および組成物 |
| 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 |
| WO2020203880A1 (ja) * | 2019-03-29 | 2020-10-08 | 田辺三菱製薬株式会社 | Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| US20120065247A1 (en) * | 2009-03-27 | 2012-03-15 | Discoverybiomed, Inc. | Modulating ires-mediated translation |
| AU2010312751B2 (en) | 2009-10-29 | 2014-07-24 | Osaka University | Bridged artificial nucleoside and nucleotide |
| US8377901B2 (en) * | 2010-07-07 | 2013-02-19 | The University Of Manitoba | Target host factors for treating viral infection |
| WO2013163404A1 (en) * | 2012-04-27 | 2013-10-31 | The Uab Research Foundation | TREATING VIRAL INFECTIONS HAVING VIRAL RNAs TRANSLATED BY A NON-IRES MEDIATED MECHANISM |
| US10377789B2 (en) | 2012-09-21 | 2019-08-13 | Osaka University | Oligonucleotide and artificial nucleoside having guanidine bridge |
| WO2015125783A1 (ja) | 2014-02-18 | 2015-08-27 | 国立大学法人大阪大学 | 架橋型ヌクレオシドおよびヌクレオチド |
| BR112020006671A2 (pt) * | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| WO2019118916A1 (en) * | 2017-12-14 | 2019-06-20 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2020158910A1 (ja) | 2019-02-01 | 2020-08-06 | 国立大学法人大阪大学 | ５'位修飾ヌクレオシドおよびそれを用いたヌクレオチド |
| EP4013870A1 (en) * | 2019-08-13 | 2022-06-22 | Alnylam Pharmaceuticals, Inc. | Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof |
-
2021
- 2021-11-05 EP EP21889279.2A patent/EP4252846A4/en active Pending
- 2021-11-05 WO PCT/JP2021/040853 patent/WO2022097727A1/ja not_active Ceased
- 2021-11-05 US US18/035,300 patent/US20240018516A1/en active Pending
- 2021-11-05 TW TW110141414A patent/TWI910261B/zh active
- 2021-11-05 CN CN202180074713.0A patent/CN116507727A/zh active Pending
- 2021-11-05 JP JP2022560831A patent/JPWO2022097727A1/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005536230A (ja) * | 2002-04-24 | 2005-12-02 | エクスプレッション ダイアグノスティクス， インコーポレイテッド | 移植拒絶を診断およびモニタリングするための方法および組成物 |
| 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 |
| WO2020203880A1 (ja) * | 2019-03-29 | 2020-10-08 | 田辺三菱製薬株式会社 | Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物 |
Non-Patent Citations (1)
| Title |
|---|
| NAT. NEUROSCI., vol. 22, JPN6025041111, 2019, pages 1383 - 1388, ISSN: 0005702707 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022097727A1 (ja) | 2022-05-12 |
| US20240018516A1 (en) | 2024-01-18 |
| EP4252846A1 (en) | 2023-10-04 |
| TW202233838A (zh) | 2022-09-01 |
| TWI910261B (zh) | 2026-01-01 |
| EP4252846A4 (en) | 2025-12-03 |
| CN116507727A (zh) | 2023-07-28 |
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