AU2020358016A1 - Chemical modifications of small interfering RNA with minimal fluorine content - Google Patents
Chemical modifications of small interfering RNA with minimal fluorine content Download PDFInfo
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- AU2020358016A1 AU2020358016A1 AU2020358016A AU2020358016A AU2020358016A1 AU 2020358016 A1 AU2020358016 A1 AU 2020358016A1 AU 2020358016 A AU2020358016 A AU 2020358016A AU 2020358016 A AU2020358016 A AU 2020358016A AU 2020358016 A1 AU2020358016 A1 AU 2020358016A1
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- modified
- oligonucleotide
- nucleotides
- antisense strand
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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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- 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
- C12N15/1137—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 against enzymes
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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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- 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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- C—CHEMISTRY; METALLURGY
- 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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- C—CHEMISTRY; METALLURGY
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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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- C—CHEMISTRY; METALLURGY
- 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
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- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/35—Nature of the modification
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- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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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
- C12N2310/531—Stem-loop; Hairpin
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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
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
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- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (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)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| US201962909278P | 2019-10-02 | 2019-10-02 | |
| US62/909,278 | 2019-10-02 | ||
| PCT/US2020/053999 WO2021067744A1 (en) | 2019-10-02 | 2020-10-02 | Chemical modifications of small interfering rna with minimal fluorine content |
Publications (1)
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| AU2020358016A1 true AU2020358016A1 (en) | 2022-04-21 |
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| AU2020358016A Abandoned AU2020358016A1 (en) | 2019-10-02 | 2020-10-02 | Chemical modifications of small interfering RNA with minimal fluorine content |
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| US (1) | US20220389430A1 (https=) |
| EP (1) | EP4038191A1 (https=) |
| JP (1) | JP2022551269A (https=) |
| KR (1) | KR20220069103A (https=) |
| CN (1) | CN114761557B (https=) |
| AU (1) | AU2020358016A1 (https=) |
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| MX (1) | MX2022004032A (https=) |
| TW (1) | TW202126809A (https=) |
| WO (1) | WO2021067744A1 (https=) |
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| PE20250400A1 (es) | 2021-04-12 | 2025-02-11 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para inhibir cetohexoquinasa |
| CA3209418A1 (en) * | 2021-04-14 | 2022-10-20 | Utsav SAXENA | Compositions and methods for modulating pnpla3 expression |
| EP4372085A4 (en) * | 2021-07-16 | 2026-01-21 | Suzhou Ribo Life Science Co Ltd | Nucleic acid, composition and conjugate, container, preparation process and use |
| EP4405368A1 (en) * | 2021-09-22 | 2024-07-31 | Sanegene Bio USA Inc. | 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides |
| WO2023069495A1 (en) * | 2021-10-19 | 2023-04-27 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides |
| JP2024543195A (ja) | 2021-12-01 | 2024-11-19 | ディセルナ ファーマシューティカルズ インコーポレイテッド | Ａｐｏｃ３発現を調節するための組成物及び方法 |
| CN119183476A (zh) * | 2022-04-26 | 2024-12-24 | 上海拓界生物医药科技有限公司 | 氘代化学修饰和包含其的寡核苷酸 |
| TW202526017A (zh) * | 2023-09-14 | 2025-07-01 | 美商Ionis製藥公司 | 用於減少ａｐｏｃｉｉｉ表現的化合物及方法 |
| WO2025191337A2 (en) * | 2024-03-11 | 2025-09-18 | Synerk Biotech Limited | Oligonucleotide terminal phosphate derivatives and method of use |
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| CA2429814C (en) | 2000-12-01 | 2014-02-18 | Thomas Tuschl | Rna interference mediating small rna molecules |
| WO2003040395A2 (en) | 2001-11-07 | 2003-05-15 | Applera Corporation | Universal nucleotides for nucleic acid analysis |
| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| WO2007030167A1 (en) | 2005-09-02 | 2007-03-15 | Nastech Pharmaceutical Company Inc. | Modification of double-stranded ribonucleic acid molecules |
| DK2341943T3 (en) | 2008-09-22 | 2019-02-25 | Dicerna Pharmaceuticals Inc | COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS |
| US8691971B2 (en) | 2008-09-23 | 2014-04-08 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference |
| US20100249214A1 (en) | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| KR101728655B1 (ko) | 2008-12-18 | 2017-04-19 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
| WO2011005860A2 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | 5' phosphate mimics |
| WO2011133871A2 (en) | 2010-04-22 | 2011-10-27 | Alnylam Pharmaceuticals, Inc. | 5'-end derivatives |
| EP2928877B1 (en) | 2012-12-06 | 2020-01-22 | Merck Sharp & Dohme Corp. | Disulfide masked prodrug compositions and methods |
| CA2950960A1 (en) | 2014-06-06 | 2015-12-10 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
| DK3569711T3 (da) | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| WO2019075419A1 (en) * | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
| UA128786C2 (uk) * | 2017-10-20 | 2024-10-23 | Дайсерна Фармасьютикалз, Інк | Олігонуклеотид для лікування інфекції гепатиту в |
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| CA3153026A1 (en) | 2021-04-08 |
| CN114761557B (zh) | 2024-11-15 |
| CL2022000825A1 (es) | 2023-01-27 |
| CN114761557A (zh) | 2022-07-15 |
| JP2022551269A (ja) | 2022-12-08 |
| US20220389430A1 (en) | 2022-12-08 |
| KR20220069103A (ko) | 2022-05-26 |
| EP4038191A1 (en) | 2022-08-10 |
| MX2022004032A (es) | 2022-09-21 |
| TW202126809A (zh) | 2021-07-16 |
| IL291841A (en) | 2022-06-01 |
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