BR112022018667A2 - Composições e métodos para inibir a expressão de ang-ptl3 - Google Patents
Composições e métodos para inibir a expressão de ang-ptl3Info
- Publication number
- BR112022018667A2 BR112022018667A2 BR112022018667A BR112022018667A BR112022018667A2 BR 112022018667 A2 BR112022018667 A2 BR 112022018667A2 BR 112022018667 A BR112022018667 A BR 112022018667A BR 112022018667 A BR112022018667 A BR 112022018667A BR 112022018667 A2 BR112022018667 A2 BR 112022018667A2
- Authority
- BR
- Brazil
- Prior art keywords
- expression
- inhibit
- compositions
- methods
- ptl3
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 102100025668 Angiopoietin-related protein 3 Human genes 0.000 abstract 4
- 101710085848 Angiopoietin-related protein 3 Proteins 0.000 abstract 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- 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 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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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- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- 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
- C12N2310/32—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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- 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
- C12N2310/3521—Methyl
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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
- C12N2310/35—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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- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Endocrinology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (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)
- Peptides Or Proteins (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062991335P | 2020-03-18 | 2020-03-18 | |
| PCT/US2021/022967 WO2021188795A1 (en) | 2020-03-18 | 2021-03-18 | Compositions and methods for inhibiting angptl3 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022018667A2 true BR112022018667A2 (pt) | 2022-11-29 |
Family
ID=75478225
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022018667A BR112022018667A2 (pt) | 2020-03-18 | 2021-03-18 | Composições e métodos para inibir a expressão de ang-ptl3 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230287425A1 (https=) |
| EP (1) | EP4121536A1 (https=) |
| JP (3) | JP7398007B2 (https=) |
| KR (1) | KR20220156880A (https=) |
| CN (1) | CN116096889A (https=) |
| AU (2) | AU2021236674B2 (https=) |
| BR (1) | BR112022018667A2 (https=) |
| CA (1) | CA3172117A1 (https=) |
| CL (1) | CL2022002521A1 (https=) |
| IL (1) | IL296549A (https=) |
| MX (1) | MX2022011550A (https=) |
| TW (2) | TWI870563B (https=) |
| WO (1) | WO2021188795A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115927335A (zh) | 2015-04-13 | 2023-04-07 | 阿尔尼拉姆医药品有限公司 | 类血管生成素3（ANGPTL3）iRNA组合物及其使用方法 |
| WO2022079222A1 (en) * | 2020-10-16 | 2022-04-21 | Sanofi | Novel rna compositions and methods for inhibiting angptl3 |
| JP2024508896A (ja) | 2021-03-04 | 2024-02-28 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびその使用方法 |
| WO2023092102A1 (en) * | 2021-11-19 | 2023-05-25 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof |
| EP4453210A1 (en) * | 2021-12-22 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors |
| US20230374522A1 (en) * | 2022-04-15 | 2023-11-23 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating scap activity |
| KR20250099265A (ko) | 2022-11-23 | 2025-07-01 | 일라이 릴리 앤드 캄파니 | 표적화 리간드-접합된 뉴클레오티드 포스포르아미다이트의 합성 방법 |
| WO2024112865A1 (en) | 2022-11-23 | 2024-05-30 | Eli Lilly And Company | Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite |
| KR20250124879A (ko) | 2022-12-22 | 2025-08-20 | 일라이 릴리 앤드 캄파니 | 올리고뉴클레오티드 단편 및 이를 사용하여 rnai 작용제를 제조하는 방법 |
| WO2024151772A2 (en) * | 2023-01-11 | 2024-07-18 | Sirius Therapeutics, Inc. | Polynucleic acid molecules for inhibiting expression of angptl3, pharmaceutical compositions, and uses thereof |
| WO2024175550A1 (en) * | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| CN118599845A (zh) * | 2023-06-27 | 2024-09-06 | 施能康医药科技(苏州)有限公司 | 靶向人血管生成素样蛋白3的核酸及其用途 |
| WO2025096970A2 (en) * | 2023-11-03 | 2025-05-08 | Basecure Therapeutics Llc | Modified oligonucleotides and methods of use thereof |
| WO2025122875A1 (en) | 2023-12-08 | 2025-06-12 | Eli Lilly And Company | Therapeutic oligonucleotide-containing pharmaceutical compositions and dosing regimens using the same |
| WO2025169133A1 (en) | 2024-02-09 | 2025-08-14 | Astrazeneca Ab | Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof |
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| US6469158B1 (en) | 1992-05-14 | 2002-10-22 | Ribozyme Pharmaceuticals, Incorporated | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| US5804683A (en) | 1992-05-14 | 1998-09-08 | Ribozyme Pharmaceuticals, Inc. | Deprotection of RNA with alkylamine |
| US5977343A (en) | 1992-05-14 | 1999-11-02 | Ribozyme Pharmaceuticals, Inc. | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| ATE227342T1 (de) | 1993-09-02 | 2002-11-15 | Ribozyme Pharm Inc | Enzymatische nukleiksaüre die nicht-nukleotide enthaltet |
| US5889136A (en) | 1995-06-09 | 1999-03-30 | The Regents Of The University Of Colorado | Orthoester protecting groups in RNA synthesis |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| US6111086A (en) | 1998-02-27 | 2000-08-29 | Scaringe; Stephen A. | Orthoester protecting groups |
| ES2336887T5 (es) | 2000-03-30 | 2019-03-06 | Whitehead Inst Biomedical Res | Mediadores de interferencia por ARN específicos de secuencias de ARN |
| CA2429814C (en) | 2000-12-01 | 2014-02-18 | Thomas Tuschl | Rna interference mediating small rna molecules |
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| KR101728655B1 (ko) | 2008-12-18 | 2017-04-19 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
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| EP2928877B1 (en) | 2012-12-06 | 2020-01-22 | Merck Sharp & Dohme Corp. | Disulfide masked prodrug compositions and methods |
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| DK3569711T3 (da) | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| US20160319278A1 (en) * | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| HUE059718T2 (hu) | 2016-09-02 | 2022-12-28 | Dicerna Pharmaceuticals Inc | 4'-foszfát analógok és azokat tartalmazó oligonukleotidok |
| EP3585895A1 (en) * | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Compositions and methods for gene editing |
| TN2020000038A1 (en) | 2017-09-14 | 2021-10-04 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use |
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2021
- 2021-03-18 US US17/906,532 patent/US20230287425A1/en active Pending
- 2021-03-18 KR KR1020227036162A patent/KR20220156880A/ko active Pending
- 2021-03-18 WO PCT/US2021/022967 patent/WO2021188795A1/en not_active Ceased
- 2021-03-18 JP JP2022556016A patent/JP7398007B2/ja active Active
- 2021-03-18 IL IL296549A patent/IL296549A/en unknown
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- 2021-03-18 MX MX2022011550A patent/MX2022011550A/es unknown
- 2021-03-18 CA CA3172117A patent/CA3172117A1/en active Pending
- 2021-03-18 TW TW110109858A patent/TWI870563B/zh active
- 2021-03-18 BR BR112022018667A patent/BR112022018667A2/pt unknown
- 2021-03-18 AU AU2021236674A patent/AU2021236674B2/en active Active
- 2021-03-18 TW TW113148812A patent/TW202530410A/zh unknown
- 2021-03-18 CN CN202180036489.6A patent/CN116096889A/zh active Pending
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Also Published As
| Publication number | Publication date |
|---|---|
| KR20220156880A (ko) | 2022-11-28 |
| CL2022002521A1 (es) | 2023-05-19 |
| JP7398007B2 (ja) | 2023-12-13 |
| JP2023518409A (ja) | 2023-05-01 |
| JP7749645B2 (ja) | 2025-10-06 |
| WO2021188795A1 (en) | 2021-09-23 |
| IL296549A (en) | 2022-11-01 |
| TWI870563B (zh) | 2025-01-21 |
| MX2022011550A (es) | 2023-01-04 |
| CA3172117A1 (en) | 2021-09-23 |
| TW202200163A (zh) | 2022-01-01 |
| EP4121536A1 (en) | 2023-01-25 |
| AU2025203784A1 (en) | 2025-06-19 |
| TW202530410A (zh) | 2025-08-01 |
| AU2021236674B2 (en) | 2025-03-06 |
| JP2026016378A (ja) | 2026-02-03 |
| AU2021236674A1 (en) | 2022-10-13 |
| CN116096889A (zh) | 2023-05-09 |
| US20230287425A1 (en) | 2023-09-14 |
| JP2024041748A (ja) | 2024-03-27 |
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