CO2020006900A2 - Medios y método para preparar vectores virales y usos de los mismos - Google Patents
Medios y método para preparar vectores virales y usos de los mismosInfo
- Publication number
- CO2020006900A2 CO2020006900A2 CONC2020/0006900A CO2020006900A CO2020006900A2 CO 2020006900 A2 CO2020006900 A2 CO 2020006900A2 CO 2020006900 A CO2020006900 A CO 2020006900A CO 2020006900 A2 CO2020006900 A2 CO 2020006900A2
- Authority
- CO
- Colombia
- Prior art keywords
- viral vectors
- preparing viral
- preparing
- compositions
- methods
- Prior art date
Links
Classifications
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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
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- 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
- A61K47/02—Inorganic compounds
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- 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
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- 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
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
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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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- 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
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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
- 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
- C12N15/1137—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 enzymes
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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
- 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
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/525—Virus
- A61K2039/5256—Virus expressing foreign proteins
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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
-
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Manufacturing & Machinery (AREA)
- Psychiatry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Inorganic Chemistry (AREA)
Abstract
Se proporcionan métodos para preparar y purificar partículas virales, y composiciones y usos que comprenden las mismas.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762583035P | 2017-11-08 | 2017-11-08 | |
PCT/US2018/058744 WO2019094253A1 (en) | 2017-11-08 | 2018-11-01 | Means and method for preparing viral vectors and uses of same |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2020006900A2 true CO2020006900A2 (es) | 2020-06-19 |
Family
ID=64362706
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2020/0006900A CO2020006900A2 (es) | 2017-11-08 | 2020-06-04 | Medios y método para preparar vectores virales y usos de los mismos |
Country Status (15)
Country | Link |
---|---|
US (1) | US20210317474A1 (es) |
EP (1) | EP3707264A1 (es) |
JP (1) | JP2021502123A (es) |
KR (1) | KR20200086292A (es) |
CN (1) | CN111566220A (es) |
AU (1) | AU2018365677A1 (es) |
BR (1) | BR112019013268A2 (es) |
CA (1) | CA3082136A1 (es) |
CL (1) | CL2020001193A1 (es) |
CO (1) | CO2020006900A2 (es) |
IL (1) | IL274430A (es) |
MX (1) | MX2020004830A (es) |
SG (1) | SG11202004406VA (es) |
TW (1) | TWI827560B (es) |
WO (1) | WO2019094253A1 (es) |
Families Citing this family (26)
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CA3069880A1 (en) * | 2017-07-17 | 2019-01-24 | Spark Therapeutics, Inc. | Apheresis methods and uses |
JP7478675B2 (ja) * | 2018-06-08 | 2024-05-07 | ノバルティス アーゲー | 薬物製品の効力を測定するための細胞ベースアッセイ |
EP3837374A4 (en) * | 2018-08-15 | 2022-06-08 | Biogen MA Inc. | COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY |
CN114174324A (zh) * | 2019-04-10 | 2022-03-11 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
US10801042B1 (en) * | 2019-07-15 | 2020-10-13 | Vigene Biosciences, Inc. | Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus |
WO2021016075A1 (en) | 2019-07-19 | 2021-01-28 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
CN114174514A (zh) | 2019-07-25 | 2022-03-11 | 诺华股份有限公司 | 可调节的表达系统 |
US20220267798A1 (en) * | 2019-08-08 | 2022-08-25 | Biogen Ma Inc. | Potency assays for viral vector production |
IL295154A (en) * | 2020-01-28 | 2022-09-01 | Lonza Walkersville Inc | Methods for determining viral titer |
WO2021209941A1 (en) | 2020-04-17 | 2021-10-21 | Friedrich Miescher Institute For Biomedical Research | Use of an orphan motif to increase expression of a heterologous transgene |
CN112121179A (zh) * | 2020-09-15 | 2020-12-25 | 山东兴瑞生物科技有限公司 | 一种组合物及其在治疗脊髓性肌萎缩症中的应用 |
CN112251440A (zh) * | 2020-10-29 | 2021-01-22 | 大连理工大学 | 一种CRISPR/Cas9基因载体、其制备方法及应用 |
JP2023552535A (ja) | 2020-12-03 | 2023-12-18 | フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ | 異種導入遺伝子の発現を制御するためのオーファンモチーフ(orphan motif)とCpG密度との組み合わせの使用 |
US20240200037A1 (en) * | 2021-04-15 | 2024-06-20 | The University Of Tokyo | Method for producing virus particles |
EP4377459A2 (en) | 2021-07-30 | 2024-06-05 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
WO2023010135A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
WO2023012514A1 (en) * | 2021-08-04 | 2023-02-09 | Takeda Pharmaceutical Company, Limited | Adeno-associated virus separation on a cation exchanger |
CN117980484A (zh) | 2021-09-16 | 2024-05-03 | 诺华股份有限公司 | 新颖的转录因子 |
CN118119710A (zh) * | 2021-10-15 | 2024-05-31 | 上海天泽云泰生物医药有限公司 | 用于治疗脊髓性肌萎缩的重组腺相关病毒载体 |
WO2023073526A1 (en) | 2021-10-25 | 2023-05-04 | Novartis Ag | Methods for improving adeno-associated virus (aav) delivery |
WO2023095034A1 (en) | 2021-11-24 | 2023-06-01 | Novartis Ag | Modulators of adeno-associated virus transduction and uses thereof |
WO2023214346A1 (en) | 2022-05-06 | 2023-11-09 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
WO2024015881A2 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | Compositions, systems, and methods for targeted transcriptional activation |
CN114990162A (zh) * | 2022-07-18 | 2022-09-02 | 苏州吉纳星辰生物技术有限公司 | 一种降低aav病毒载体生产中质粒残留的方法 |
WO2024027632A1 (en) * | 2022-08-01 | 2024-02-08 | Skyline Therapeutics (Shanghai) Co., Ltd. | Novel plasmid backbone to reduce dna impurities in raav preparation |
CN115554418B (zh) * | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
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WO2017160360A2 (en) * | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
JP7364306B2 (ja) * | 2016-03-31 | 2023-10-18 | スパーク セラピューティクス インコーポレイテッド | カラムに基づく高度にスケーラブルなrAAVの製造プロセス |
KR20230125339A (ko) * | 2016-04-15 | 2023-08-29 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
-
2018
- 2018-11-01 CA CA3082136A patent/CA3082136A1/en active Pending
- 2018-11-01 KR KR1020207014865A patent/KR20200086292A/ko not_active Application Discontinuation
- 2018-11-01 EP EP18804830.0A patent/EP3707264A1/en active Pending
- 2018-11-01 MX MX2020004830A patent/MX2020004830A/es unknown
- 2018-11-01 JP JP2020544560A patent/JP2021502123A/ja active Pending
- 2018-11-01 BR BR112019013268A patent/BR112019013268A2/pt not_active Application Discontinuation
- 2018-11-01 SG SG11202004406VA patent/SG11202004406VA/en unknown
- 2018-11-01 AU AU2018365677A patent/AU2018365677A1/en active Pending
- 2018-11-01 CN CN201880085757.1A patent/CN111566220A/zh active Pending
- 2018-11-01 US US16/761,869 patent/US20210317474A1/en active Pending
- 2018-11-01 TW TW107138852A patent/TWI827560B/zh active
- 2018-11-01 WO PCT/US2018/058744 patent/WO2019094253A1/en active Application Filing
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2020
- 2020-05-04 IL IL274430A patent/IL274430A/en unknown
- 2020-05-06 CL CL2020001193A patent/CL2020001193A1/es unknown
- 2020-06-04 CO CONC2020/0006900A patent/CO2020006900A2/es unknown
Also Published As
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TWI827560B (zh) | 2024-01-01 |
SG11202004406VA (en) | 2020-06-29 |
TW201930590A (zh) | 2019-08-01 |
KR20200086292A (ko) | 2020-07-16 |
BR112019013268A2 (pt) | 2019-12-17 |
MX2020004830A (es) | 2020-11-11 |
US20210317474A1 (en) | 2021-10-14 |
CL2020001193A1 (es) | 2020-11-13 |
JP2021502123A (ja) | 2021-01-28 |
WO2019094253A1 (en) | 2019-05-16 |
CA3082136A1 (en) | 2019-05-16 |
EP3707264A1 (en) | 2020-09-16 |
AU2018365677A1 (en) | 2020-05-14 |
CN111566220A (zh) | 2020-08-21 |
RU2020118557A (ru) | 2021-12-08 |
RU2020118557A3 (es) | 2022-04-15 |
IL274430A (en) | 2020-06-30 |
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