AU2019257708B2 - Expression of human FOXP3 in gene edited T cells - Google Patents
Expression of human FOXP3 in gene edited T cells Download PDFInfo
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- AU2019257708B2 AU2019257708B2 AU2019257708A AU2019257708A AU2019257708B2 AU 2019257708 B2 AU2019257708 B2 AU 2019257708B2 AU 2019257708 A AU2019257708 A AU 2019257708A AU 2019257708 A AU2019257708 A AU 2019257708A AU 2019257708 B2 AU2019257708 B2 AU 2019257708B2
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- foxp3
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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
- A61K35/14—Blood; Artificial blood
- A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
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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
- A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
- A61K40/22—Immunosuppressive or immunotolerising
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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
- A61K40/416—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
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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
- A61K40/418—Antigens related to induction of tolerance to non-self
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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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
- C12N15/63—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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
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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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- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/60—Transcription factors
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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
- C12N2510/00—Genetically modified cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Developmental Biology & Embryology (AREA)
- Mycology (AREA)
- Transplantation (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2024266825A AU2024266825A1 (en) | 2018-04-27 | 2024-11-22 | Expression of human FOXP3 in gene edited T cells |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862663561P | 2018-04-27 | 2018-04-27 | |
| US62/663,561 | 2018-04-27 | ||
| US201862773414P | 2018-11-30 | 2018-11-30 | |
| US62/773,414 | 2018-11-30 | ||
| PCT/US2019/029159 WO2019210078A1 (en) | 2018-04-27 | 2019-04-25 | Expression of human foxp3 in gene edited t cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024266825A Division AU2024266825A1 (en) | 2018-04-27 | 2024-11-22 | Expression of human FOXP3 in gene edited T cells |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2019257708A1 AU2019257708A1 (en) | 2020-09-03 |
| AU2019257708B2 true AU2019257708B2 (en) | 2024-08-22 |
Family
ID=68295504
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019257708A Active AU2019257708B2 (en) | 2018-04-27 | 2019-04-25 | Expression of human FOXP3 in gene edited T cells |
| AU2024266825A Pending AU2024266825A1 (en) | 2018-04-27 | 2024-11-22 | Expression of human FOXP3 in gene edited T cells |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024266825A Pending AU2024266825A1 (en) | 2018-04-27 | 2024-11-22 | Expression of human FOXP3 in gene edited T cells |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20210253652A1 (enExample) |
| EP (1) | EP3784689A4 (enExample) |
| JP (2) | JP7575949B2 (enExample) |
| KR (1) | KR20210005146A (enExample) |
| CN (1) | CN112041334A (enExample) |
| AU (2) | AU2019257708B2 (enExample) |
| CA (1) | CA3091491A1 (enExample) |
| IL (1) | IL277036A (enExample) |
| SG (1) | SG11202007877SA (enExample) |
| WO (1) | WO2019210078A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2016427822B2 (en) * | 2016-10-31 | 2025-01-23 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene |
| CN112218882A (zh) | 2018-04-27 | 2021-01-12 | 西雅图儿童医院(Dba西雅图儿童研究所) | Foxp3在经编辑的cd34+细胞中的表达 |
| EP3921427A1 (en) * | 2019-02-05 | 2021-12-15 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Recombinant vectors suitable for the treatment of ipex syndrome |
| CA3141159A1 (en) * | 2019-05-21 | 2020-11-26 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| EP4055039A1 (en) * | 2019-11-08 | 2022-09-14 | Sangamo Therapeutics, Inc. | Generation of engineered regulatory t cells |
| WO2021163642A2 (en) * | 2020-02-13 | 2021-08-19 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients |
| TW202227631A (zh) * | 2020-11-09 | 2022-07-16 | 英商圭爾醫療有限公司 | 冷凍保存經工程化之調節t細胞(tregs)之方法 |
| EP4355879A4 (en) * | 2021-06-14 | 2025-07-23 | Kamau Therapeutics Inc | METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS |
| WO2023122099A2 (en) * | 2021-12-21 | 2023-06-29 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr-based gene editing to preserve splicing and expression of foxp3 isoforms 1 and 2 |
| CN115305237B (zh) * | 2022-07-08 | 2023-07-14 | 中南大学 | 一种优化的调节性t细胞的扩增及规模化生产的方法 |
| EP4634383A1 (en) * | 2022-12-16 | 2025-10-22 | Life Edit Therapeutics, Inc. | Guide rnas that target foxp3 gene and methods of use |
| WO2025006409A2 (en) * | 2023-06-24 | 2025-01-02 | Sonoma Biotherapeutics, Inc. | Compositions and methods for engineering cells |
| WO2025054503A1 (en) * | 2023-09-08 | 2025-03-13 | Sonoma Biotherapeutics, Inc. | Cells ectopically expressing foxp3 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110123502A1 (en) | 2007-02-21 | 2011-05-26 | Barry Simon C | Method for obtaining treg-cells |
| ES2883131T3 (es) * | 2013-05-29 | 2021-12-07 | Cellectis | Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN |
| ES2880473T5 (es) * | 2015-01-30 | 2024-05-09 | Univ California | Suministro de proteínas en células hematopoyéticas primarias |
| HK1254190A1 (zh) * | 2015-05-08 | 2019-07-12 | President And Fellows Of Harvard College | 通用供体干细胞和相关方法 |
| CA3034094A1 (en) | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| WO2018073391A1 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Targeted gene insertion for improved immune cells therapy |
| WO2018081476A2 (en) * | 2016-10-27 | 2018-05-03 | Intima Bioscience, Inc. | Viral methods of t cell therapy |
| AU2016427822B2 (en) | 2016-10-31 | 2025-01-23 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene |
| CN109790518A (zh) * | 2017-05-08 | 2019-05-21 | 中国科学院动物研究所 | 经修饰的t细胞、其制备方法及用途 |
| EP3672617A4 (en) * | 2017-08-22 | 2021-06-23 | The Regents of the University of California | LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES |
| CN112218882A (zh) | 2018-04-27 | 2021-01-12 | 西雅图儿童医院(Dba西雅图儿童研究所) | Foxp3在经编辑的cd34+细胞中的表达 |
-
2019
- 2019-04-25 US US16/981,213 patent/US20210253652A1/en active Pending
- 2019-04-25 KR KR1020207033790A patent/KR20210005146A/ko active Pending
- 2019-04-25 WO PCT/US2019/029159 patent/WO2019210078A1/en not_active Ceased
- 2019-04-25 CN CN201980028523.8A patent/CN112041334A/zh active Pending
- 2019-04-25 CA CA3091491A patent/CA3091491A1/en active Pending
- 2019-04-25 EP EP19792759.3A patent/EP3784689A4/en active Pending
- 2019-04-25 AU AU2019257708A patent/AU2019257708B2/en active Active
- 2019-04-25 JP JP2020560338A patent/JP7575949B2/ja active Active
- 2019-04-25 SG SG11202007877SA patent/SG11202007877SA/en unknown
-
2020
- 2020-08-31 IL IL277036A patent/IL277036A/en unknown
-
2024
- 2024-10-18 JP JP2024182444A patent/JP2025013879A/ja active Pending
- 2024-11-22 AU AU2024266825A patent/AU2024266825A1/en active Pending
Non-Patent Citations (2)
| Title |
|---|
| GOODWIN, M., et al, 2016, '123. Gene Editing as a Therapeutic Approach to Treat IPEX Syndrome", Molecular Therapy, 24, pages S51-S52 * |
| KORNETE, M., et al., 2018, 'Highly efficient and versatile plasmid-based gene editing in primary T cells", The Journal of Immunology, 200(7), pages 2489-2501 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2019210078A1 (en) | 2019-10-31 |
| IL277036A (en) | 2020-10-29 |
| US20210253652A1 (en) | 2021-08-19 |
| JP7575949B2 (ja) | 2024-10-31 |
| KR20210005146A (ko) | 2021-01-13 |
| SG11202007877SA (en) | 2020-09-29 |
| JP2021521849A (ja) | 2021-08-30 |
| CA3091491A1 (en) | 2019-10-31 |
| AU2019257708A1 (en) | 2020-09-03 |
| EP3784689A1 (en) | 2021-03-03 |
| JP2025013879A (ja) | 2025-01-28 |
| AU2024266825A1 (en) | 2024-12-12 |
| CN112041334A (zh) | 2020-12-04 |
| EP3784689A4 (en) | 2022-01-19 |
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