CN119193702A - 经由改造的adar募集的rna和dna碱基编辑 - Google Patents
经由改造的adar募集的rna和dna碱基编辑 Download PDFInfo
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- CN119193702A CN119193702A CN202410647165.1A CN202410647165A CN119193702A CN 119193702 A CN119193702 A CN 119193702A CN 202410647165 A CN202410647165 A CN 202410647165A CN 119193702 A CN119193702 A CN 119193702A
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- rna
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- nucleic acid
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- C—CHEMISTRY; METALLURGY
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/16—Aptamers
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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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14341—Use of virus, viral particle or viral elements as a vector
- C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Virology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
Applications Claiming Priority (12)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862728007P | 2018-09-06 | 2018-09-06 | |
| US62/728,007 | 2018-09-06 | ||
| US201862766433P | 2018-10-17 | 2018-10-17 | |
| US62/766,433 | 2018-10-17 | ||
| US201862773150P | 2018-11-29 | 2018-11-29 | |
| US201862773146P | 2018-11-29 | 2018-11-29 | |
| US62/773,146 | 2018-11-29 | ||
| US62/773,150 | 2018-11-29 | ||
| US201862780241P | 2018-12-15 | 2018-12-15 | |
| US62/780,241 | 2018-12-15 | ||
| CN201980072798.1A CN112996912A (zh) | 2018-09-06 | 2019-09-06 | 经由改造的adar募集的rna和dna碱基编辑 |
| PCT/US2019/050095 WO2020051555A1 (en) | 2018-09-06 | 2019-09-06 | Rna and dna base editing via engneered adar recruitment |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980072798.1A Division CN112996912A (zh) | 2018-09-06 | 2019-09-06 | 经由改造的adar募集的rna和dna碱基编辑 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN119193702A true CN119193702A (zh) | 2024-12-27 |
Family
ID=69723301
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202410647165.1A Pending CN119193702A (zh) | 2018-09-06 | 2019-09-06 | 经由改造的adar募集的rna和dna碱基编辑 |
| CN201980072798.1A Pending CN112996912A (zh) | 2018-09-06 | 2019-09-06 | 经由改造的adar募集的rna和dna碱基编辑 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980072798.1A Pending CN112996912A (zh) | 2018-09-06 | 2019-09-06 | 经由改造的adar募集的rna和dna碱基编辑 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20220010333A1 (https=) |
| EP (2) | EP4332224A3 (https=) |
| JP (1) | JP7720623B6 (https=) |
| CN (2) | CN119193702A (https=) |
| AU (1) | AU2019336793B2 (https=) |
| CA (1) | CA3110998A1 (https=) |
| GB (1) | GB2590880A (https=) |
| WO (1) | WO2020051555A1 (https=) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110612353A (zh) * | 2017-03-03 | 2019-12-24 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| WO2019104094A2 (en) | 2017-11-21 | 2019-05-31 | The Regents Of The University Of California | Fusion proteins and methods for site-directed genome editing |
| EP3956449B1 (en) | 2019-04-15 | 2026-02-25 | EdiGene Therapeutics (Beijing) Inc. | Methods and compositions for editing rnas |
| EP3997229A4 (en) * | 2019-07-12 | 2024-07-03 | Peking University | TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA |
| WO2021113264A1 (en) * | 2019-12-02 | 2021-06-10 | The Regents Of The University Of California | Engineering circular guide rnas |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| US20230059753A1 (en) * | 2019-12-09 | 2023-02-23 | Astellas Pharma Inc. | Antisense guide rna with added functional region for editing target rna |
| CN115777020A (zh) * | 2020-04-22 | 2023-03-10 | 塑造治疗公司 | 使用snrna组分的组合物和方法 |
| EP4158019A1 (en) * | 2020-05-26 | 2023-04-05 | Shape Therapeutics Inc. | Methods and compositions relating to engineered guide systems for adenosine deaminase acting on rna editing |
| JP2023528039A (ja) * | 2020-05-26 | 2023-07-03 | シェイプ セラピューティクス インコーポレイテッド | 改変環状ポリヌクレオチド |
| JP2023529316A (ja) * | 2020-05-26 | 2023-07-10 | シェイプ セラピューティクス インコーポレイテッド | ゲノム編集のための組成物及び方法 |
| US20230272379A1 (en) * | 2020-07-06 | 2023-08-31 | Edigene Therapeutics (Beijing ) Inc. | Improved rna editing method |
| CN116209762A (zh) * | 2020-07-30 | 2023-06-02 | 阿达尔克斯制药有限公司 | Adar依赖性编辑组合物及其使用方法 |
| WO2022046667A1 (en) * | 2020-08-24 | 2022-03-03 | Wave Life Sciences Ltd. | Cells and non-human animals engineered to express adar1 and uses thereof |
| WO2022078569A1 (en) * | 2020-10-12 | 2022-04-21 | Eberhard Karls Universität Tübingen | Artificial nucleic acids for rna editing |
| JP2024500279A (ja) * | 2020-11-11 | 2024-01-09 | シェイプ セラピューティクス インコーポレイテッド | Rna編集組成物及び使用方法 |
| EP4256050A4 (en) * | 2020-12-01 | 2025-01-22 | Shape Therapeutics Inc. | High throughput rna-editing screening methods |
| US20240150754A1 (en) * | 2020-12-25 | 2024-05-09 | Astellas Pharma Inc. | Guide rna for editing polyadenylation signal sequence of target rna |
| CA3220338A1 (en) * | 2021-05-25 | 2022-12-01 | Yiannis SAVVA | Engineered guide rnas and polynucleotides |
| WO2023278436A1 (en) * | 2021-06-29 | 2023-01-05 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| EP4388089A4 (en) * | 2021-08-18 | 2025-01-15 | Peking University | Engineered adar-recruiting rnas and methods of use thereof |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| JP2023040852A (ja) * | 2021-09-10 | 2023-03-23 | 隆光 矢野 | -1フレームシフトを誘導するための1本鎖核酸分子及び組成物 |
| US20230167441A1 (en) * | 2021-11-30 | 2023-06-01 | Minghong Zhong | Segmented Nucleic Acids |
| US20250115908A1 (en) * | 2022-02-07 | 2025-04-10 | The Regents Of The University Of California | Method of interfering with repetitive rna |
| WO2023196853A1 (en) * | 2022-04-05 | 2023-10-12 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of muscular dystrophies |
| CN116732039B (zh) * | 2023-08-03 | 2023-11-14 | 呈诺再生医学科技(北京)有限公司 | 一种促进rna序列在细胞内直接环化翻译的序列组合及其应用 |
| CN117210435A (zh) * | 2023-08-29 | 2023-12-12 | 苏州湃芮生物科技有限公司 | 一种用于调控rna甲基化修饰的编辑系统及其应用 |
| CN121059628A (zh) * | 2024-06-03 | 2025-12-05 | 时夕(广州)生物科技有限公司 | 调控RNA剪接的向导agRNA |
| WO2026080897A1 (en) * | 2024-10-11 | 2026-04-16 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of chronic pain |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| FR2764387B1 (fr) * | 1997-06-05 | 1999-07-23 | Centre Nat Rech Scient | Utilisation d'une proteine fluorescente pour la detection d'interactions entre une proteine cible et son ligand |
| MX367100B (es) * | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| EP3234134B1 (en) * | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| CA3005245A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of alagille syndrome |
| CN109642231A (zh) * | 2016-06-17 | 2019-04-16 | 博德研究所 | Vi型crispr直向同源物和系统 |
| WO2017220751A1 (en) * | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| WO2018208998A1 (en) * | 2017-05-10 | 2018-11-15 | The Regents Of The University Of California | Directed editing of cellular rna via nuclear delivery of crispr/cas9 |
-
2019
- 2019-09-06 US US17/273,885 patent/US20220010333A1/en not_active Abandoned
- 2019-09-06 WO PCT/US2019/050095 patent/WO2020051555A1/en not_active Ceased
- 2019-09-06 GB GB2104889.7A patent/GB2590880A/en not_active Withdrawn
- 2019-09-06 JP JP2021511542A patent/JP7720623B6/ja active Active
- 2019-09-06 CA CA3110998A patent/CA3110998A1/en active Pending
- 2019-09-06 EP EP23214540.9A patent/EP4332224A3/en active Pending
- 2019-09-06 CN CN202410647165.1A patent/CN119193702A/zh active Pending
- 2019-09-06 EP EP19857915.3A patent/EP3847255A4/en not_active Withdrawn
- 2019-09-06 AU AU2019336793A patent/AU2019336793B2/en active Active
- 2019-09-06 CN CN201980072798.1A patent/CN112996912A/zh active Pending
-
2025
- 2025-07-07 US US19/261,672 patent/US20260092292A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3110998A1 (en) | 2020-03-12 |
| JP7720623B2 (ja) | 2025-08-08 |
| EP4332224A2 (en) | 2024-03-06 |
| EP3847255A4 (en) | 2024-01-10 |
| EP3847255A1 (en) | 2021-07-14 |
| AU2019336793A1 (en) | 2021-04-08 |
| AU2019336793B2 (en) | 2024-11-21 |
| US20260092292A1 (en) | 2026-04-02 |
| GB202104889D0 (en) | 2021-05-19 |
| US20220010333A1 (en) | 2022-01-13 |
| EP4332224A3 (en) | 2024-06-05 |
| WO2020051555A1 (en) | 2020-03-12 |
| JP2021536244A (ja) | 2021-12-27 |
| GB2590880A (en) | 2021-07-07 |
| JP7720623B6 (ja) | 2025-09-18 |
| CN112996912A (zh) | 2021-06-18 |
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