HK1248723B - Gene therapy - Google Patents

Gene therapy Download PDF

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Publication number
HK1248723B
HK1248723B HK18108313.3A HK18108313A HK1248723B HK 1248723 B HK1248723 B HK 1248723B HK 18108313 A HK18108313 A HK 18108313A HK 1248723 B HK1248723 B HK 1248723B
Authority
HK
Hong Kong
Prior art keywords
aav
aav vector
factor
cfi
pharmaceutical composition
Prior art date
Application number
HK18108313.3A
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German (de)
English (en)
French (fr)
Chinese (zh)
Other versions
HK1248723A1 (en
Inventor
GROENDAHL Christian
Funnell Tim
Hollowood Chris
Original Assignee
Syncona IP Holdco Limited
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Publication date
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=55130363&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1248723(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Syncona IP Holdco Limited filed Critical Syncona IP Holdco Limited
Publication of HK1248723A1 publication Critical patent/HK1248723A1/en
Publication of HK1248723B publication Critical patent/HK1248723B/en

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    • 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
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1725Complement proteins, e.g. anaphylatoxin, C3a or C5a
    • 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
    • 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
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525Virus
    • A61K2039/5258Virus-like particles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/60Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6031Proteins
    • A61K2039/6075Viral proteins
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
HK18108313.3A 2015-10-28 2016-10-27 Gene therapy HK1248723B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1519086.1A GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy
GB1519086.1 2015-10-28
PCT/GB2016/053343 WO2017072515A1 (en) 2015-10-28 2016-10-27 Gene therapy

Publications (2)

Publication Number Publication Date
HK1248723A1 HK1248723A1 (en) 2018-10-19
HK1248723B true HK1248723B (en) 2019-11-22

Family

ID=55130363

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18108313.3A HK1248723B (en) 2015-10-28 2016-10-27 Gene therapy

Country Status (19)

Country Link
US (3) US20190255193A1 (enExample)
EP (3) EP3262066B2 (enExample)
JP (3) JP7126944B2 (enExample)
KR (1) KR20180066247A (enExample)
CN (4) CN116271104A (enExample)
AU (3) AU2016344523B2 (enExample)
CA (1) CA3002125A1 (enExample)
DK (2) DK3262066T4 (enExample)
ES (2) ES2666204T5 (enExample)
GB (1) GB201519086D0 (enExample)
HK (1) HK1248723B (enExample)
IL (3) IL284577B2 (enExample)
MX (1) MX366343B (enExample)
NO (1) NO3262066T3 (enExample)
PL (2) PL3262066T5 (enExample)
RU (1) RU2740038C2 (enExample)
SG (1) SG11201802934WA (enExample)
WO (1) WO2017072515A1 (enExample)
ZA (1) ZA201802462B (enExample)

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WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
GB201608046D0 (en) * 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
EP4445954A3 (en) * 2017-07-31 2025-01-08 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
AU2018351491A1 (en) * 2017-10-20 2020-05-07 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration
WO2020077091A1 (en) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Kir 7.1 gene therapy vectors and methods of using the same
WO2020086735A1 (en) * 2018-10-23 2020-04-30 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
EP4667578A2 (en) * 2019-04-03 2025-12-24 REGENXBIO Inc. Gene therapy for eye pathologies
US20220313722A1 (en) * 2019-09-09 2022-10-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb
CA3182800A1 (en) 2020-06-14 2021-12-23 Vertex Pharmaceuticals Inc. Complement factor i-related compositions and methods
GB202009741D0 (en) 2020-06-25 2020-08-12 Freeline Therapeutics Ltd Polynucleotide
WO2022056531A1 (en) * 2020-09-09 2022-03-17 Homology Medicines, Inc. Vectorized antibodies and uses thereof
US20230390296A1 (en) 2020-10-30 2023-12-07 Keio University Novel treatment and prevention of sarcopenia-related diseases
GB202018320D0 (en) * 2020-11-20 2021-01-06 Univ Newcastle Methods of producing recombinant complement proteins
TW202323270A (zh) 2021-11-04 2023-06-16 英商自由行療法有限公司 多肽
US20250161255A1 (en) 2022-01-25 2025-05-22 Motoshi Hayano New therapy and prevention for suppressing aging-related disorders including sarcopenia

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ES2611608T3 (es) * 2005-10-21 2017-05-09 Catalyst Biosciences, Inc. Proteasas modificadas que inhiben la activación del complemento
JP5250548B2 (ja) 2006-06-21 2013-07-31 エムユーエスシー ファウンデーション フォー リサーチ ディベロップメント 疾患の治療のための補体h因子の標的化
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JP5332064B2 (ja) 2007-03-01 2013-11-06 ウェルスタット イムノセラピューティクス, エルエルシー 炎症により特徴付けられる疾患の治療
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
GB0816702D0 (en) * 2008-09-12 2008-10-22 Trinity College Dublin Complement proteins
GB0904427D0 (en) 2009-03-13 2009-04-29 Lachmann Peter Treatment of diseases related to hyperactivity of the complement system
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
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Publication number Publication date
BR112018008731A2 (en) 2018-11-06
KR20180066247A (ko) 2018-06-18
MX366343B (es) 2019-07-05
JP7126944B2 (ja) 2022-08-29
CN116370655A (zh) 2023-07-04
SG11201802934WA (en) 2018-05-30
ES2828050T3 (es) 2021-05-25
DK3262066T4 (da) 2023-08-21
IL284577B2 (en) 2023-12-01
PL3262066T5 (pl) 2023-10-30
EP3262066B2 (en) 2023-06-07
IL258806A (en) 2018-06-28
MX2018005145A (es) 2018-08-09
PL3262066T3 (pl) 2018-07-31
US20230277689A1 (en) 2023-09-07
JP2024156706A (ja) 2024-11-06
ES2666204T3 (es) 2018-05-03
RU2018118954A (ru) 2019-12-02
NO3262066T3 (enExample) 2018-07-07
GB201519086D0 (en) 2015-12-09
DK3262066T3 (en) 2018-05-22
RU2740038C2 (ru) 2020-12-30
CN117343960A (zh) 2024-01-05
RU2018118954A3 (enExample) 2020-03-13
EP3262066A1 (en) 2018-01-03
CN108431030A (zh) 2018-08-21
PL3360890T3 (pl) 2020-12-28
AU2016344523A1 (en) 2018-06-14
EP3360890A1 (en) 2018-08-15
AU2022203675B2 (en) 2025-03-06
IL258806B (en) 2021-07-29
AU2025200979A1 (en) 2025-03-06
EP3262066B1 (en) 2018-02-07
BR112018008731A8 (pt) 2020-03-31
US20250319210A1 (en) 2025-10-16
EP3360890B1 (en) 2020-10-07
AU2022203675A1 (en) 2022-06-16
IL304084A (en) 2023-08-01
JP2022141670A (ja) 2022-09-29
CN116271104A (zh) 2023-06-23
US20190255193A1 (en) 2019-08-22
CA3002125A1 (en) 2017-05-04
ES2666204T5 (es) 2023-11-03
DK3360890T3 (da) 2020-11-02
WO2017072515A1 (en) 2017-05-04
IL304084B1 (en) 2025-11-01
ZA201802462B (en) 2021-01-27
IL284577A (en) 2021-08-31
CN108431030B (zh) 2023-10-20
EP3786176A1 (en) 2021-03-03
AU2016344523B2 (en) 2022-03-17
JP2018537122A (ja) 2018-12-20
IL284577B1 (en) 2023-08-01

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