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RS64769B1 - Varijante adeno-asociranih virusa i postupci upotrebe - Google Patents

Varijante adeno-asociranih virusa i postupci upotrebe

Info

Publication number
RS64769B1
RS64769B1 RS20231029A RSP20231029A RS64769B1 RS 64769 B1 RS64769 B1 RS 64769B1 RS 20231029 A RS20231029 A RS 20231029A RS P20231029 A RSP20231029 A RS P20231029A RS 64769 B1 RS64769 B1 RS 64769B1
Authority
RS
Serbia
Prior art keywords
methods
associated viruses
variant adeno
adeno
variant
Prior art date
Application number
RS20231029A
Other languages
English (en)
Inventor
Joshua Dudman
Adam Hantman
Loren Looger
Kimberly Ritola
David Schaffer
Dougal Gowanlock Robinson Tervo
Sarada Viswanathan
Bum-Yeol Hwang
Alla Karpova
Original Assignee
Hughes Howard Med Inst
Univ California
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 Hughes Howard Med Inst, Univ California filed Critical Hughes Howard Med Inst
Publication of RS64769B1 publication Critical patent/RS64769B1/sr

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B30/00Methods of screening libraries
    • C40B30/06Methods of screening libraries by measuring effects on living organisms, tissues or cells
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00Libraries per se, e.g. arrays, mixtures
    • C40B40/04Libraries containing only organic compounds
    • C40B40/06Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
RS20231029A 2016-06-15 2017-06-15 Varijante adeno-asociranih virusa i postupci upotrebe RS64769B1 (sr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662350361P 2016-06-15 2016-06-15
US201662404585P 2016-10-05 2016-10-05
PCT/US2017/037781 WO2017218842A1 (en) 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using
EP17734575.8A EP3472183B1 (en) 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using

Publications (1)

Publication Number Publication Date
RS64769B1 true RS64769B1 (sr) 2023-11-30

Family

ID=59258370

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20231029A RS64769B1 (sr) 2016-06-15 2017-06-15 Varijante adeno-asociranih virusa i postupci upotrebe

Country Status (27)

Country Link
US (2) US10961282B2 (sr)
EP (2) EP3472183B1 (sr)
JP (2) JP7094277B2 (sr)
KR (2) KR20230054754A (sr)
CN (2) CN109641939B (sr)
AU (2) AU2017286652B2 (sr)
BR (1) BR112018076090A2 (sr)
CA (1) CA3028113A1 (sr)
DK (1) DK3472183T5 (sr)
EA (1) EA201990033A1 (sr)
ES (1) ES2962611T3 (sr)
FI (1) FI3472183T3 (sr)
HR (1) HRP20231403T1 (sr)
HU (1) HUE063755T2 (sr)
IL (2) IL263719B2 (sr)
LT (1) LT3472183T (sr)
MA (1) MA44546B1 (sr)
MX (2) MX2018015770A (sr)
MY (1) MY190221A (sr)
PH (1) PH12018502664A1 (sr)
PL (1) PL3472183T3 (sr)
PT (1) PT3472183T (sr)
RS (1) RS64769B1 (sr)
SG (1) SG11201811189RA (sr)
SI (1) SI3472183T1 (sr)
WO (1) WO2017218842A1 (sr)
ZA (1) ZA201900278B (sr)

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US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
CA3023592C (en) * 2016-05-13 2020-12-08 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018022905A2 (en) 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
AU2018324477A1 (en) 2017-08-28 2019-10-17 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
CN111601884A (zh) * 2017-10-16 2020-08-28 维格内罗有限责任公司 Aav载体
CA3108324A1 (en) * 2018-07-31 2020-02-06 Cornell University Gene therapy methods to control organ function
CA3114199A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021022208A1 (en) * 2019-08-01 2021-02-04 Kaplitt Michael G Targeted gene therapy to treat neurological diseases
US20230265453A1 (en) * 2020-07-08 2023-08-24 Baylor College Of Medicine Gene therapy for stxbp1 encephalopathy
CN111825772B (zh) * 2020-07-30 2023-10-20 中国科学院精密测量科学与技术创新研究院 具有变异衣壳蛋白的腺相关病毒及其应用
US20230277687A1 (en) 2020-08-06 2023-09-07 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
AU2021401998A1 (en) * 2020-12-16 2023-08-03 Children's Medical Research Institute Adeno-associated virus capsids and vectors
WO2022126188A1 (en) * 2020-12-16 2022-06-23 Children's Medical Research Institute Aav capsids and vectors
BR112023026523A2 (pt) 2021-06-17 2024-03-05 Meiragtx Uk Ii Ltd Métodos para fabricar aav
EP4370538A1 (en) 2021-07-14 2024-05-22 Meiragtx UK II Limited Kcnv2 gene therapy
WO2023288184A2 (en) * 2021-07-14 2023-01-19 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
CA3225084A1 (en) 2021-07-14 2023-01-19 MEIRAGTX, UK II Limited Retgc gene therapy
WO2023131811A2 (en) 2021-12-15 2023-07-13 Meiragtx Uk Ii Limited Polycistronic expression of gut peptides
WO2023192450A1 (en) * 2022-03-30 2023-10-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
WO2024059667A2 (en) * 2022-09-13 2024-03-21 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
WO2024079655A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Chromatography methods for purification of aav capsids
WO2024079530A2 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Nucleic acid regulatory elements for gene expression in the muscle and methods of use
WO2024079661A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Atp7b gene therapy
WO2024079657A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Formulations for aav gene therapy
WO2024079662A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Upf1 expression constructs
WO2024126696A1 (en) 2022-12-14 2024-06-20 King's College London Compositions and methods for treating neurological diseases
WO2024148189A2 (en) * 2023-01-05 2024-07-11 Emugen Therapeutics Llc Neuronal promoters and uses thereof
WO2024148179A2 (en) * 2023-01-05 2024-07-11 Emugen Therapeutics Llc Retro-aav and use in treating neurodegenerative diseases
WO2024161022A2 (en) 2023-02-03 2024-08-08 King's College London Compositions and methods for treating neurological diseases
WO2024213660A1 (en) 2023-04-13 2024-10-17 Meiragtx Uk Ii Limited Inflammation-inducible promoters

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CA3023592C (en) * 2016-05-13 2020-12-08 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018022905A2 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Also Published As

Publication number Publication date
PL3472183T3 (pl) 2024-02-12
MA44546B1 (fr) 2021-03-31
US20210230231A1 (en) 2021-07-29
HRP20231403T1 (hr) 2024-02-16
PT3472183T (pt) 2023-11-16
KR102522661B1 (ko) 2023-04-18
EP4268852A3 (en) 2024-02-07
LT3472183T (lt) 2023-09-25
FI3472183T3 (fi) 2023-11-03
PH12018502664A1 (en) 2019-10-07
IL263719A (en) 2019-01-31
MX2018015770A (es) 2019-08-29
SI3472183T1 (sl) 2023-11-30
EP4268852A2 (en) 2023-11-01
KR20190039930A (ko) 2019-04-16
JP2019518793A (ja) 2019-07-04
CN109641939B (zh) 2022-11-08
MY190221A (en) 2022-04-06
JP7579301B2 (ja) 2024-11-07
ES2962611T3 (es) 2024-03-20
SG11201811189RA (en) 2019-01-30
IL263719B2 (en) 2023-09-01
EP3472183B1 (en) 2023-08-09
AU2017286652A1 (en) 2019-01-24
CN116003532A (zh) 2023-04-25
US20190300579A1 (en) 2019-10-03
MX2024000505A (es) 2024-03-19
JP7094277B2 (ja) 2022-07-01
EA201990033A1 (ru) 2019-06-28
US10961282B2 (en) 2021-03-30
HUE063755T2 (hu) 2024-01-28
WO2017218842A1 (en) 2017-12-21
BR112018076090A2 (pt) 2019-03-26
IL300981A (en) 2023-04-01
IL263719B1 (en) 2023-05-01
KR20230054754A (ko) 2023-04-25
JP2022126759A (ja) 2022-08-30
DK3472183T5 (da) 2024-09-09
AU2017286652B2 (en) 2021-10-07
AU2021225247A1 (en) 2021-09-30
CA3028113A1 (en) 2017-12-21
ZA201900278B (en) 2022-04-28
MA44546A1 (fr) 2019-07-31
EP3472183A1 (en) 2019-04-24
AU2021225247B2 (en) 2023-02-16
US11939355B2 (en) 2024-03-26
CN109641939A (zh) 2019-04-16
DK3472183T3 (da) 2023-11-06

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