CA3085338A1 - Cpf1-related methods and compositions for gene editing - Google Patents
Cpf1-related methods and compositions for gene editing Download PDFInfo
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- CA3085338A1 CA3085338A1 CA3085338A CA3085338A CA3085338A1 CA 3085338 A1 CA3085338 A1 CA 3085338A1 CA 3085338 A CA3085338 A CA 3085338A CA 3085338 A CA3085338 A CA 3085338A CA 3085338 A1 CA3085338 A1 CA 3085338A1
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
- A61K40/4202—Receptors, cell surface antigens or cell surface determinants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
- A61K40/4202—Receptors, cell surface antigens or cell surface determinants
- A61K40/421—Immunoglobulin superfamily
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
- C12N5/0637—Immunosuppressive T lymphocytes, e.g. regulatory T cells or Treg
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
- C12Q1/44—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase involving esterase
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2121/00—Preparations for use in therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2510/00—Genetically modified cells
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
- Medicinal Chemistry (AREA)
- Developmental Biology & Embryology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Analytical Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762597118P | 2017-12-11 | 2017-12-11 | |
| US62/597,118 | 2017-12-11 | ||
| US201862623501P | 2018-01-29 | 2018-01-29 | |
| US62/623,501 | 2018-01-29 | ||
| US201862664905P | 2018-04-30 | 2018-04-30 | |
| US62/664,905 | 2018-04-30 | ||
| US201862746494P | 2018-10-16 | 2018-10-16 | |
| US62/746,494 | 2018-10-16 | ||
| PCT/US2018/065032 WO2019118516A1 (en) | 2017-12-11 | 2018-12-11 | Cpf1-related methods and compositions for gene editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3085338A1 true CA3085338A1 (en) | 2019-06-20 |
Family
ID=65023985
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3085338A Pending CA3085338A1 (en) | 2017-12-11 | 2018-12-11 | Cpf1-related methods and compositions for gene editing |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20200299661A1 (enExample) |
| EP (1) | EP3724326A1 (enExample) |
| JP (2) | JP2021505187A (enExample) |
| KR (1) | KR20200097760A (enExample) |
| CN (1) | CN111712569A (enExample) |
| AU (1) | AU2018383712B2 (enExample) |
| CA (1) | CA3085338A1 (enExample) |
| MX (1) | MX2020006072A (enExample) |
| WO (1) | WO2019118516A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111647618A (zh) * | 2020-01-15 | 2020-09-11 | 温州医科大学 | 一种新型基因组编辑工具(Lb2Cas12a-RVR)及其构建方法和应用方法 |
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| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| SG11202008956XA (en) * | 2018-03-14 | 2020-10-29 | Editas Medicine Inc | Systems and methods for the treatment of hemoglobinopathies |
| US12133884B2 (en) | 2018-05-11 | 2024-11-05 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US20200216825A1 (en) * | 2019-01-08 | 2020-07-09 | Integrated Dna Technologies, Inc. | CAS12a MUTANT GENES AND POLYPEPTIDES ENCODED BY SAME |
| AU2020223060B2 (en) * | 2019-02-13 | 2023-04-13 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| EP4047087A4 (en) * | 2019-08-19 | 2023-08-23 | Southern Medical University | Construction of high-fidelity crispr/ascpf1 mutant and application thereof |
| JP7696335B2 (ja) * | 2019-09-09 | 2025-06-20 | スクライブ・セラピューティクス・インコーポレイテッド | 免疫療法に使用するための組成物および方法 |
| US20230009232A1 (en) * | 2019-11-20 | 2023-01-12 | Cartherics Pty. Ltd. | Method for providing immune cells with enhanced function |
| CN113355389B (zh) * | 2020-03-05 | 2022-11-15 | 广西扬翔股份有限公司 | 利用CRISPR/Cas12a系统靶向富集核酸目标区域的方法及其应用 |
| EP4146800A1 (en) * | 2020-05-08 | 2023-03-15 | Metagenomi, Inc. | Enzymes with ruvc domains |
| KR20220005208A (ko) * | 2020-07-06 | 2022-01-13 | 주식회사 지씨셀 | 면역원성이 감소된 신규한 이식용 세포 |
| KR102497690B1 (ko) * | 2020-09-22 | 2023-02-10 | (주)지플러스생명과학 | 신규한 crispr 연관 단백질 및 이의 용도 |
| US20230374478A1 (en) * | 2020-09-22 | 2023-11-23 | G Flas Life Sciences | Modified cas12a protein and use thereof |
| US20230416732A1 (en) * | 2020-10-30 | 2023-12-28 | Arbor Biotechnologies, Inc. | Compositions comprising an rna guide targeting bcl11a and uses thereof |
| AU2021369840A1 (en) * | 2020-10-30 | 2023-06-01 | Arbor Biotechnologies, Inc. | Compositions comprising an rna guide targeting b2m and uses thereof |
| US20240026351A1 (en) * | 2020-10-30 | 2024-01-25 | Arbor Biotechnologies, Inc. | Compositions comprising an rna guide targeting trac and uses thereof |
| WO2022103935A1 (en) * | 2020-11-11 | 2022-05-19 | The Trustees Of Columbia University In The City Of New York | Multiplex epigenome editing |
| US11591381B2 (en) | 2020-11-30 | 2023-02-28 | Crispr Therapeutics Ag | Gene-edited natural killer cells |
| AU2021392032A1 (en) | 2020-12-03 | 2023-06-22 | Century Therapeutics, Inc. | Genetically engineered cells and uses thereof |
| US11661459B2 (en) | 2020-12-03 | 2023-05-30 | Century Therapeutics, Inc. | Artificial cell death polypeptide for chimeric antigen receptor and uses thereof |
| AU2021396403A1 (en) * | 2020-12-11 | 2023-06-29 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class ii in a cell |
| EP4259160A4 (en) * | 2020-12-14 | 2025-05-14 | Emendobio Inc. | BIALLELIC INACTIVATION OF B2M |
| KR20230135068A (ko) * | 2020-12-23 | 2023-09-22 | 인텔리아 테라퓨틱스, 인크. | 세포에서 ciita를 유전적으로 변형시키기 위한 조성물및 방법 |
| EP4271798A1 (en) | 2020-12-30 | 2023-11-08 | CRISPR Therapeutics AG | Compositions and methods for differentiating stem cells into nk cells |
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| KR102787489B1 (ko) * | 2021-06-16 | 2025-03-31 | 한국생명공학연구원 | enCpf1 및 키메릭 DNA-RNA 가이드를 포함하는 유전체 교정 또는 발현 억제용 조성물 |
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| US20250122534A1 (en) | 2021-08-02 | 2025-04-17 | Vor Biopharma Inc. | Compositions and methods for gene modification |
| WO2023028348A1 (en) | 2021-08-27 | 2023-03-02 | Metagenomi, Inc. | Enzymes with ruvc domains |
| US20240417755A1 (en) | 2021-09-27 | 2024-12-19 | Vor Biopharma Inc. | Fusion polypeptides for genetic editing and methods of use thereof |
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| CN116144631B (zh) * | 2023-01-17 | 2023-09-15 | 华中农业大学 | 耐热型核酸内切酶及其介导的基因编辑系统 |
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| EP4427809A3 (en) * | 2014-10-31 | 2024-12-04 | The Trustees of The University of Pennsylvania | Altering gene expression in car-t cells and uses thereof |
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2018
- 2018-12-11 EP EP18834094.7A patent/EP3724326A1/en active Pending
- 2018-12-11 AU AU2018383712A patent/AU2018383712B2/en active Active
- 2018-12-11 MX MX2020006072A patent/MX2020006072A/es unknown
- 2018-12-11 JP JP2020531964A patent/JP2021505187A/ja active Pending
- 2018-12-11 CA CA3085338A patent/CA3085338A1/en active Pending
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- 2018-12-11 WO PCT/US2018/065032 patent/WO2019118516A1/en not_active Ceased
- 2018-12-11 CN CN201880089010.3A patent/CN111712569A/zh active Pending
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2023
- 2023-11-13 JP JP2023193282A patent/JP2024023294A/ja active Pending
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111647618A (zh) * | 2020-01-15 | 2020-09-11 | 温州医科大学 | 一种新型基因组编辑工具(Lb2Cas12a-RVR)及其构建方法和应用方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| CN111712569A (zh) | 2020-09-25 |
| WO2019118516A1 (en) | 2019-06-20 |
| AU2018383712A1 (en) | 2020-07-02 |
| AU2018383712B2 (en) | 2025-09-04 |
| MX2020006072A (es) | 2020-08-24 |
| JP2021505187A (ja) | 2021-02-18 |
| EP3724326A1 (en) | 2020-10-21 |
| JP2024023294A (ja) | 2024-02-21 |
| US20200299661A1 (en) | 2020-09-24 |
| KR20200097760A (ko) | 2020-08-19 |
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