JP2024513972A - Rnaサイレンシング剤および使用方法 - Google Patents
Rnaサイレンシング剤および使用方法 Download PDFInfo
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- JP2024513972A JP2024513972A JP2023562698A JP2023562698A JP2024513972A JP 2024513972 A JP2024513972 A JP 2024513972A JP 2023562698 A JP2023562698 A JP 2023562698A JP 2023562698 A JP2023562698 A JP 2023562698A JP 2024513972 A JP2024513972 A JP 2024513972A
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- 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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- 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
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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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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
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- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163174507P | 2021-04-13 | 2021-04-13 | |
| US63/174,507 | 2021-04-13 | ||
| PCT/US2022/024692 WO2022221457A1 (en) | 2021-04-13 | 2022-04-13 | Rna silencing agents and methods of use |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024513972A true JP2024513972A (ja) | 2024-03-27 |
| JP2024513972A5 JP2024513972A5 (enExample) | 2025-04-09 |
Family
ID=83639690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023562698A Pending JP2024513972A (ja) | 2021-04-13 | 2022-04-13 | Rnaサイレンシング剤および使用方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240191229A1 (enExample) |
| EP (1) | EP4323376A4 (enExample) |
| JP (1) | JP2024513972A (enExample) |
| CN (1) | CN117425664A (enExample) |
| AU (1) | AU2022257002A1 (enExample) |
| CA (1) | CA3216732A1 (enExample) |
| WO (1) | WO2022221457A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| US20240401045A1 (en) * | 2021-10-01 | 2024-12-05 | Adarx Pharmaceuticals, Inc. | Angiotensinogen-modulating compositions and methods of use thereof |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012512651A (ja) * | 2008-12-18 | 2012-06-07 | ダイセルナ ファーマシューティカルズ, インコーポレイテッド | 遺伝子発現の特異的阻害のための拡張dicer基質薬剤および方法 |
| JP2013535982A (ja) * | 2010-08-24 | 2013-09-19 | メルク・シャープ・エンド・ドーム・コーポレイション | 内部非核酸スペーサーを含む一本鎖RNAi剤関連出願の相互参照本出願は、2010年8月24日に出願された米国特許仮出願第61/376,471号の恩典を主張し、該仮出願は引用により本明細書に組み込まれる。 |
| WO2016011123A1 (en) * | 2014-07-16 | 2016-01-21 | Arrowhead Research Corporation | Organic compositions to treat apoc3-related diseases |
| JP2017537618A (ja) * | 2014-12-17 | 2017-12-21 | プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップProQR Therapeutics II B.V. | 標的化rna編集 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8188060B2 (en) * | 2008-02-11 | 2012-05-29 | Dharmacon, Inc. | Duplex oligonucleotides with enhanced functionality in gene regulation |
| EP2264169A1 (en) * | 2009-06-15 | 2010-12-22 | Qiagen GmbH | Modified siNA |
| KR101696704B1 (ko) * | 2013-12-17 | 2017-01-16 | 주식회사 인코드젠 | 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도 |
-
2022
- 2022-04-13 JP JP2023562698A patent/JP2024513972A/ja active Pending
- 2022-04-13 CN CN202280034139.0A patent/CN117425664A/zh active Pending
- 2022-04-13 WO PCT/US2022/024692 patent/WO2022221457A1/en not_active Ceased
- 2022-04-13 EP EP22788889.8A patent/EP4323376A4/en active Pending
- 2022-04-13 CA CA3216732A patent/CA3216732A1/en active Pending
- 2022-04-13 AU AU2022257002A patent/AU2022257002A1/en active Pending
- 2022-04-13 US US18/286,640 patent/US20240191229A1/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012512651A (ja) * | 2008-12-18 | 2012-06-07 | ダイセルナ ファーマシューティカルズ, インコーポレイテッド | 遺伝子発現の特異的阻害のための拡張dicer基質薬剤および方法 |
| JP2013535982A (ja) * | 2010-08-24 | 2013-09-19 | メルク・シャープ・エンド・ドーム・コーポレイション | 内部非核酸スペーサーを含む一本鎖RNAi剤関連出願の相互参照本出願は、2010年8月24日に出願された米国特許仮出願第61/376,471号の恩典を主張し、該仮出願は引用により本明細書に組み込まれる。 |
| WO2016011123A1 (en) * | 2014-07-16 | 2016-01-21 | Arrowhead Research Corporation | Organic compositions to treat apoc3-related diseases |
| JP2017537618A (ja) * | 2014-12-17 | 2017-12-21 | プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップProQR Therapeutics II B.V. | 標的化rna編集 |
Non-Patent Citations (4)
| Title |
|---|
| HUANG HUANG ET AL, NUCLEIC ACIDS RESEARCH, vol. 37, no. 22, JPN7026001114, 2009, pages 7560 - 7569, ISSN: 0005818139 * |
| NA WEI ET AL, PLOS ONE, vol. Vol. 7, Issue 11, Article number e49309, JPN7026001115, 2012, ISSN: 0005818140 * |
| QUAN DU ET AL, NUCLEIC ACIDS RESEARCH, vol. 33, no. 5, JPN7026001113, 2005, pages 1671 - 1677, ISSN: 0005818138 * |
| TOBY PASSIOURA ET AL, BMC BIOTECHNOLOGY, vol. Vol. 9, Article number 57, JPN7026001116, 2009, ISSN: 0005818141 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CN117425664A (zh) | 2024-01-19 |
| US20240191229A1 (en) | 2024-06-13 |
| CA3216732A1 (en) | 2022-10-20 |
| EP4323376A4 (en) | 2025-07-09 |
| WO2022221457A1 (en) | 2022-10-20 |
| AU2022257002A1 (en) | 2023-10-26 |
| EP4323376A1 (en) | 2024-02-21 |
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