EA201892285A1 - Нуклеотиды, модифицированные циклофосфонатом по положению 5' - Google Patents
Нуклеотиды, модифицированные циклофосфонатом по положению 5'Info
- Publication number
- EA201892285A1 EA201892285A1 EA201892285A EA201892285A EA201892285A1 EA 201892285 A1 EA201892285 A1 EA 201892285A1 EA 201892285 A EA201892285 A EA 201892285A EA 201892285 A EA201892285 A EA 201892285A EA 201892285 A1 EA201892285 A1 EA 201892285A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- cyclophosphonate
- nucleotides modified
- stranded
- regulation
- oligonucleotides
- Prior art date
Links
- 239000002773 nucleotide Substances 0.000 title abstract 5
- 125000003729 nucleotide group Chemical group 0.000 abstract 4
- 239000003795 chemical substances by application Substances 0.000 abstract 3
- 230000009368 gene silencing by RNA Effects 0.000 abstract 3
- 108091034117 Oligonucleotide Proteins 0.000 abstract 2
- 108091030071 RNAI Proteins 0.000 abstract 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 2
- 230000014509 gene expression Effects 0.000 abstract 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 238000012790 confirmation Methods 0.000 abstract 1
- 238000003745 diagnosis Methods 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001629 suppression Effects 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
- C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
- C07H19/067—Pyrimidine radicals with ribosyl as the saccharide radical
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H1/00—Processes for the preparation of sugar derivatives
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
- C07H19/167—Purine radicals with ribosyl as the saccharide radical
-
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
- C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
- C07H19/207—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids the phosphoric or polyphosphoric acids being esterified by a further hydroxylic compound, e.g. flavine adenine dinucleotide or nicotinamide-adenine dinucleotide
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- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C12N15/09—Recombinant DNA-technology
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
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- 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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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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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
- C12N2310/3519—Fusion with another nucleic acid
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- Chemical & Material Sciences (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
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- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
Abstract
Описаны нуклеотиды, модифицированные циклофосфонатом по положению 5', и олигонуклеотиды, такие как агенты РНК-интерференции (РНКи), содержащие нуклеотиды, модифицированные циклофосфонатом по положению 5'. Агенты РНКи, содержащие двухцепочечные или одноцепочечные олигонуклеотиды, описанные в настоящем документе, содержащие нуклеотиды, модифицированные циклофосфонатом по положению 5', подходят для модуляции экспрессии гена, а также применения в терапии, диагностике, подтверждении мишени и изучении генома. Агенты РНКи и одноцепочечные антисмысловые олигонуклеотиды, содержащие нуклеотиды, модифицированные циклофосфонатом по положению 5', подходят для лечения заболеваний или состояний, при которых имеется ответ на подавление экспрессии или активности гена в клетке, ткани или организме.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662346304P | 2016-06-06 | 2016-06-06 | |
PCT/US2017/036108 WO2017214112A1 (en) | 2016-06-06 | 2017-06-06 | 5'-cyclo-phosphonate modified nucleotides |
Publications (1)
Publication Number | Publication Date |
---|---|
EA201892285A1 true EA201892285A1 (ru) | 2019-07-31 |
Family
ID=60579042
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201892285A EA201892285A1 (ru) | 2016-06-06 | 2017-06-06 | Нуклеотиды, модифицированные циклофосфонатом по положению 5' |
Country Status (12)
Country | Link |
---|---|
US (2) | US11078227B2 (ru) |
EP (1) | EP3464313A4 (ru) |
JP (2) | JP7028865B2 (ru) |
KR (2) | KR102639586B1 (ru) |
CN (2) | CN114736256A (ru) |
AU (3) | AU2017279512B2 (ru) |
CA (1) | CA3023764A1 (ru) |
EA (1) | EA201892285A1 (ru) |
IL (2) | IL263437B (ru) |
MX (2) | MX2018015109A (ru) |
TW (1) | TWI815794B (ru) |
WO (1) | WO2017214112A1 (ru) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
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FI3607069T3 (fi) | 2017-04-05 | 2023-01-13 | Tuotteita ja koostumuksia | |
JP2022535588A (ja) * | 2019-06-06 | 2022-08-09 | アビディティー バイオサイエンシーズ,インク. | 核酸ポリペプチド組成物およびその使用 |
EP4211243A2 (en) | 2020-09-11 | 2023-07-19 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
AU2021401424A1 (en) | 2020-12-18 | 2023-06-29 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating factor xii |
CA3227661A1 (en) | 2021-08-05 | 2023-02-09 | Weimin Wang | 1'-alkyl modified ribose derivatives and methods of use |
KR20240082361A (ko) | 2021-09-22 | 2024-06-10 | 사네진 바이오 유에스에이 인크. | 올리고뉴클레오티드의 생체내 전달에 사용하기 위한 2'-알킬 또는 3'-알킬 변형된 리보스 유도체 |
US20230227480A1 (en) | 2021-10-05 | 2023-07-20 | Sanegene Bio Usa Inc. | Polyhydroxylated cyclopentane derivatives and methods of use |
US20230346819A1 (en) | 2022-02-22 | 2023-11-02 | Sanegene Bio Usa Inc. | 5'-modified carbocyclic ribonucleotide derivatives and methods of use |
WO2023220561A1 (en) | 2022-05-09 | 2023-11-16 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
WO2023241587A1 (zh) * | 2022-06-14 | 2023-12-21 | 大睿生物医药科技(上海)有限公司 | 环膦酸酯修饰的核苷酸 |
WO2023245060A2 (en) | 2022-06-15 | 2023-12-21 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use |
US20240218363A1 (en) | 2022-07-11 | 2024-07-04 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
US20240052348A1 (en) | 2022-08-05 | 2024-02-15 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiotensinogen (agt) and methods of use thereof |
WO2024062413A1 (en) * | 2022-09-21 | 2024-03-28 | Janssen Biotech, Inc. | Novel stabilized nucleoside phosphates and analogues thereof |
US20240175026A1 (en) | 2022-10-14 | 2024-05-30 | Sanegene Bio Usa Inc. | Small interfering rna targeting c3 and uses thereof |
WO2024097674A2 (en) * | 2022-11-02 | 2024-05-10 | Aligos Therapeutics, Inc. | Modified short interfering nucleic acid (sina) molecules and uses thereof |
WO2024137590A2 (en) | 2022-12-19 | 2024-06-27 | Sanegene Bio Usa Inc. | Small interfering rna targeting cfb and uses thereof |
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GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
ES2086997T3 (es) | 1993-01-25 | 1996-07-01 | Hybridon Inc | Oligonucleotido-alquilfosfonatos y -alquilfosfonotioatos. |
CA2159632A1 (en) | 1993-03-31 | 1994-10-13 | Ashis Kumar Saha | Novel 5'-substituted nucleosides and oligomers produced therefrom |
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2017
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- 2017-06-06 JP JP2019516096A patent/JP7028865B2/ja active Active
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- 2017-06-06 CN CN202210360585.2A patent/CN114736256A/zh active Pending
- 2017-06-06 TW TW106118757A patent/TWI815794B/zh active
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2018
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2022
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2023
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TWI815794B (zh) | 2023-09-21 |
IL290633A (en) | 2022-04-01 |
MX2018015109A (es) | 2019-04-22 |
CA3023764A1 (en) | 2017-12-14 |
TW201801748A (zh) | 2018-01-16 |
JP7028865B2 (ja) | 2022-03-02 |
KR102639586B1 (ko) | 2024-02-23 |
JP2019517588A (ja) | 2019-06-24 |
CN114736256A (zh) | 2022-07-12 |
WO2017214112A1 (en) | 2017-12-14 |
KR102426487B1 (ko) | 2022-07-27 |
AU2021266265A1 (en) | 2021-12-09 |
EP3464313A4 (en) | 2020-02-26 |
IL263437B (en) | 2022-03-01 |
AU2023275805A1 (en) | 2024-02-22 |
AU2017279512B2 (en) | 2021-08-19 |
AU2017279512A1 (en) | 2018-12-06 |
IL290633B1 (en) | 2024-07-01 |
KR20190015277A (ko) | 2019-02-13 |
KR20220108204A (ko) | 2022-08-02 |
US11078227B2 (en) | 2021-08-03 |
US20220119439A1 (en) | 2022-04-21 |
CN109526222A (zh) | 2019-03-26 |
CN109526222B (zh) | 2022-04-29 |
IL263437A (en) | 2019-01-31 |
JP2022059052A (ja) | 2022-04-12 |
EP3464313A1 (en) | 2019-04-10 |
US20190085012A1 (en) | 2019-03-21 |
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