MA51353A1 - Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit - Google Patents

Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

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Publication number
MA51353A1
MA51353A1 MA51353A MA51353A MA51353A1 MA 51353 A1 MA51353 A1 MA 51353A1 MA 51353 A MA51353 A MA 51353A MA 51353 A MA51353 A MA 51353A MA 51353 A1 MA51353 A1 MA 51353A1
Authority
MA
Morocco
Prior art keywords
hybrid
aavrh74
aav9
associated virus
recombinant adeno
Prior art date
Application number
MA51353A
Other languages
English (en)
Other versions
MA51353B1 (fr
Inventor
Giuseppe Ronzitti
Isabelle Richard
Evelyne Gicquel
Frederico Mingozzi
Patrice Vidal
Original Assignee
Univ Sorbonne
Genethon
Inst Nat Sante Rech Med
Univ Devry Val Dessonne
Association Inst De Myologie
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Sorbonne, Genethon, Inst Nat Sante Rech Med, Univ Devry Val Dessonne, Association Inst De Myologie filed Critical Univ Sorbonne
Publication of MA51353A1 publication Critical patent/MA51353A1/fr
Publication of MA51353B1 publication Critical patent/MA51353B1/fr

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
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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
    • C12N15/111—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
    • C12N15/63—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • 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 RNAses, DNAses
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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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14145—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne une protéine de la capside du virus adéno-associé (aav) recombinant, qui est un hybride entre le sérotype 9 d'aav (aav9) et des protéines de la capside de sérotype 74 d'aav (aavrh74), ladite protéine de la capside d'aav hybride recombinante présentant un tropisme hépatique réduit par rapport aux protéines de la capside d'aav9 et d'aavrh74 parents. L'invention concerne également les particules de vecteur de sérotype d'aav hybrides dérivées qui conditionnent un gène d'intérêt et leur utilisation en thérapie génique, en particulier pour le traitement de maladies génétiques neuromusculaires.
MA51353A 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit MA51353B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18305399 2018-04-05
PCT/EP2019/058560 WO2019193119A1 (fr) 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Publications (2)

Publication Number Publication Date
MA51353A1 true MA51353A1 (fr) 2021-09-30
MA51353B1 MA51353B1 (fr) 2022-09-30

Family

ID=62063468

Family Applications (1)

Application Number Title Priority Date Filing Date
MA51353A MA51353B1 (fr) 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Country Status (13)

Country Link
US (3) US12037362B2 (fr)
EP (2) EP3775173A1 (fr)
JP (2) JP7374119B2 (fr)
KR (2) KR20210015770A (fr)
CN (2) CN112004925B (fr)
AU (2) AU2019249890B2 (fr)
BR (2) BR112020020223A2 (fr)
CA (2) CA3093347A1 (fr)
IL (2) IL277740A (fr)
MA (1) MA51353B1 (fr)
TN (1) TN2020000187A1 (fr)
WO (2) WO2019193119A1 (fr)
ZA (1) ZA202005422B (fr)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3097375A1 (fr) * 2018-04-27 2019-10-31 Universitat Heidelberg Polypeptides de capside d'aav modifies pour le traitement de maladies musculaires
CA3149449A1 (fr) 2019-09-19 2021-03-25 Evelyne GICQUEL Systeme d'expression de therapie genique attenuant la toxicite cardiaque de fkrp
EP4073087A4 (fr) * 2019-11-08 2023-11-29 The Board Of Regents Of The University Of Texas System Vecteur viral adéno-associé recombiné pour la délivrance de gènes
EP4073257A1 (fr) * 2019-12-09 2022-10-19 UCL Business Ltd Composition de thérapie génique et traitement de cardiomyopathie liée à la myh7
JP2023507460A (ja) 2019-12-18 2023-02-22 ジェネトン 植物毛状根からの組換えウイルスベクターの生産
WO2021229255A1 (fr) 2020-05-14 2021-11-18 Genethon Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps
EP3913060A1 (fr) 2020-05-22 2021-11-24 Genethon Vecteurs codant la glucose-6-phosphatase (g6pase-a) pour la thérapie génique
US20230265427A1 (en) 2020-06-09 2023-08-24 Genethon Treatment of Genetic Dilated Cardiomyopathies
EP4162049A1 (fr) 2020-06-09 2023-04-12 Genethon Inhibiteurs de cilp-1 destinés à être utilisés dans le traitement de cardiomyopathies dilatées
WO2021250081A1 (fr) 2020-06-09 2021-12-16 Genethon Activateur de nrf2 destiné à être utilisé dans le traitement de cardiomyopathies dilatées
EP4168052A2 (fr) 2020-06-19 2023-04-26 Genethon Système d'expression de thérapie génique permettant une expression adéquate dans les muscles et dans le coeur de sgcg
CA3187635A1 (fr) 2020-07-03 2022-01-06 Genethon Procede d'ingenierie de nouveaux capsides aav hybrides par l'intermediaire d'une permutation de regions hypervariables
CN116194587A (zh) 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
WO2022053630A1 (fr) 2020-09-10 2022-03-17 Genethon Capside de aav modifiée par peptide
EP4222263A1 (fr) 2020-09-29 2023-08-09 Genethon Amélioration de l'expression d'utrophine dans une cellule par induction de mutations dans des éléments régulateurs d'utrophine et leur utilisation thérapeutique
CN116635088A (zh) * 2020-12-29 2023-08-22 江苏金斯瑞蓬勃生物科技有限公司 高分散性的hek293t细胞株及其筛选方法
WO2022204476A1 (fr) 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Édition de nucléotides pour remettre en phase des transcrits de la dmd par édition de base et édition génomique prémium (« prime editing »)
WO2022226301A1 (fr) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Capsides de vaa tropiques chimériques cardiaques
WO2023012313A1 (fr) 2021-08-04 2023-02-09 Genethon Promoteurs hybrides pour l'expression génique dans les muscles et dans le snc
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
AU2023211652A1 (en) 2022-01-25 2024-08-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
CN114657152A (zh) * 2022-03-31 2022-06-24 苏州博腾生物制药有限公司 一种新型aav血清型及其制备方法
WO2023237731A1 (fr) 2022-06-09 2023-12-14 Genethon Gde tronquée au niveau du terminal n pour le traitement de la maladie de stockage du glycogène de type iii
WO2023237748A1 (fr) 2022-06-10 2023-12-14 Genethon Capside d'aav modifiée par un peptide présentant une efficacité de transduction musculaire améliorée
WO2024074727A1 (fr) 2022-10-07 2024-04-11 Genethon Immunothérapie cellulaire par car-t anti-fap pour traiter des myopathies squelettiques
WO2024079249A1 (fr) 2022-10-12 2024-04-18 Genethon Vecteur aav hybride améliorant l'expression d'un transgène dans le foie
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024173802A2 (fr) * 2023-02-17 2024-08-22 Biogen Ma Inc. Peptides contenant rgd à des fins de distribution de charges utiles
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024188913A1 (fr) 2023-03-10 2024-09-19 Genethon Induction de la tolérance immunitaire par un vecteur aav comprenant la combinaison d'une capside ciblant le foie et d'un promoteur tandem spécifique du foie et des muscles
CN118005749B (zh) * 2023-07-21 2024-07-16 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN117031014A (zh) * 2023-08-14 2023-11-10 陕西脉元生物科技有限公司 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5054975B2 (ja) 2003-09-30 2012-10-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途
CN117363655A (zh) 2005-04-07 2024-01-09 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
WO2013123503A1 (fr) 2012-02-17 2013-08-22 The Children's Hospital Of Philadelphia Compositions de vecteurs de virus adéno-associés et méthodes de transfert de gènes dans des cellules, des organes et des tissus
US9909142B2 (en) 2012-04-18 2018-03-06 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
EP2660325A3 (fr) 2012-05-02 2014-02-12 Christian Medical College Vecteurs de virus AAV et séquences de nucléotides et procédés correspondants
BR112015021884A8 (pt) * 2013-03-15 2019-11-26 Childrens Hospital Philadelphia vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica
MX2015014712A (es) * 2013-04-20 2016-04-27 Res Inst Nationwide Childrens Hospital Administración de virus adeno-asociado recombinante de construcciones de polinucléotidos u7snrna dirigida al exon 2.
RU2705249C2 (ru) * 2013-07-12 2019-11-06 Дзе Чилдрен'З Хоспитал Оф Филадельфия Вектор aav и анализ на нейтрализующие антитела против aav (аденоассоциированного вируса)
KR102380265B1 (ko) 2013-07-22 2022-03-29 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
SG10201902574RA (en) 2014-09-24 2019-04-29 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2017096164A1 (fr) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Nouvelles capsides du virus adéno-associé (aav) recombinant offrant un tropisme amélioré des muscles squelettiques humains
EP3429605A4 (fr) 2016-03-18 2019-09-18 The Children's Hospital of Philadelphia Agent thérapeutique pour le traitement de maladies, notamment les maladies touchant le système nerveux central
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
CA3094465A1 (fr) * 2018-04-03 2019-10-10 Stridebio, Inc. Vecteurs de virus permettant de cibler des tissus ophtalmiques

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US12037362B2 (en) 2024-07-16
KR20210153047A (ko) 2021-12-16
CA3134183A1 (fr) 2020-10-08
AU2019249890B2 (en) 2024-10-10
US20210107948A1 (en) 2021-04-15
CN112004925A (zh) 2020-11-27
AU2019439673A1 (en) 2021-10-07
CA3093347A1 (fr) 2019-10-10
WO2020200499A1 (fr) 2020-10-08
BR112021019821A2 (pt) 2021-12-07
RU2020136195A (ru) 2022-05-06
EP3775173A1 (fr) 2021-02-17
BR112020020223A2 (pt) 2021-01-19
JP7374119B2 (ja) 2023-11-06
ZA202005422B (en) 2023-03-29
IL277740A (en) 2020-11-30
EP3947653A1 (fr) 2022-02-09
KR20210015770A (ko) 2021-02-10
US20240317814A1 (en) 2024-09-26
IL286926A (en) 2021-10-31
WO2019193119A1 (fr) 2019-10-10
JP2021520203A (ja) 2021-08-19
AU2019249890A1 (en) 2020-09-24
CN113795574A (zh) 2021-12-14
JP2022526405A (ja) 2022-05-24
MA51353B1 (fr) 2022-09-30
US20220228167A1 (en) 2022-07-21
CN112004925B (zh) 2024-09-27
TN2020000187A1 (en) 2022-04-04

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