KR102922606B1 - 유전자 요법 - Google Patents

유전자 요법

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Publication number
KR102922606B1
KR102922606B1 KR1020187015024A KR20187015024A KR102922606B1 KR 102922606 B1 KR102922606 B1 KR 102922606B1 KR 1020187015024 A KR1020187015024 A KR 1020187015024A KR 20187015024 A KR20187015024 A KR 20187015024A KR 102922606 B1 KR102922606 B1 KR 102922606B1
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South Korea
Prior art keywords
eye
aav
aav vector
subject
factor
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KR1020187015024A
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English (en)
Korean (ko)
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KR20180066247A (ko
Inventor
크리스티안 그뢴달
팀 퍼넬
크리스 할로우드
Original Assignee
신코나 아이피 홀드코 리미티드
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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=KR102922606(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 신코나 아이피 홀드코 리미티드 filed Critical 신코나 아이피 홀드코 리미티드
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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1725—Complement proteins, e.g. anaphylatoxin, C3a or C5a
    • 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
    • A61K48/0008—Medicinal 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
    • 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
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation 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
    • 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
    • A61K48/0075—Medicinal 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
    • 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
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • 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
    • A61K2039/5258—Virus-like particles
    • 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
    • A61K2039/60—Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6031—Proteins
    • A61K2039/6075—Viral proteins
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector 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)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Emergency Medicine (AREA)
KR1020187015024A 2015-10-28 2016-10-27 유전자 요법 Active KR102922606B1 (ko)

Applications Claiming Priority (3)

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

Publications (2)

Publication Number Publication Date
KR20180066247A KR20180066247A (ko) 2018-06-18
KR102922606B1 true KR102922606B1 (ko) 2026-02-03

Family

ID=55130363

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020187015024A Active KR102922606B1 (ko) 2015-10-28 2016-10-27 유전자 요법

Country Status (19)

Country Link
US (3) US20190255193A1 (https=)
EP (3) EP3360890B1 (https=)
JP (3) JP7126944B2 (https=)
KR (1) KR102922606B1 (https=)
CN (4) CN117343960A (https=)
AU (3) AU2016344523B2 (https=)
CA (1) CA3002125A1 (https=)
DK (2) DK3262066T4 (https=)
ES (2) ES2828050T3 (https=)
GB (1) GB201519086D0 (https=)
HK (1) HK1248723B (https=)
IL (3) IL304084B2 (https=)
MX (1) MX366343B (https=)
NO (1) NO3262066T3 (https=)
PL (2) PL3360890T3 (https=)
RU (1) RU2740038C2 (https=)
SG (1) SG11201802934WA (https=)
WO (1) WO2017072515A1 (https=)
ZA (1) ZA201802462B (https=)

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CN108291216B (zh) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
JP7046828B2 (ja) 2016-04-15 2022-04-04 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 滲出型加齢性黄斑変性の治療のための組成物
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
KR20200036912A (ko) * 2017-07-31 2020-04-07 리플렉션 바이오테크놀러지스 리미티드 안과 질환을 위한 세포 모델 및 치료요법
CN111788311A (zh) * 2017-10-20 2020-10-16 双子治疗公司 用于治疗年龄相关性黄斑变性的组合物和方法
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet 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
CA3117551A1 (en) * 2018-10-23 2020-04-30 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
EP4667578A3 (en) * 2019-04-03 2026-04-01 REGENXBIO Inc. Gene therapy for eye pathologies
EP4028025A4 (en) * 2019-09-09 2023-07-19 University of Pittsburgh - of the Commonwealth System of Higher Education METHODS OF RESTORING LYSOSOMAL FUNCTION OF RETINAL PIGMENT EPITHELIAL CELLS BY TFEB ACTIVATION
AU2021293174A1 (en) 2020-06-14 2023-01-19 Vertex Pharmaceuticals Incorporated Complement Factor I-related compositions and methods
GB202009741D0 (en) 2020-06-25 2020-08-12 Freeline Therapeutics Ltd Polynucleotide
IL301131A (en) * 2020-09-09 2023-05-01 Homology Medicines Inc Vector antibodies and their uses
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
WO2023079301A1 (en) 2021-11-04 2023-05-11 Freeline Therapeutics Limited Assays using mutant complement factor i variants with decreased activity
JPWO2023145735A1 (https=) 2022-01-25 2023-08-03

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WO2010103291A2 (en) * 2009-03-13 2010-09-16 Cambridge Enterprise Limited Treatment of diseases related to hyperactivity of the complement system

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Publication number Publication date
KR20180066247A (ko) 2018-06-18
JP2018537122A (ja) 2018-12-20
EP3262066B1 (en) 2018-02-07
NO3262066T3 (https=) 2018-07-07
HK1248723B (en) 2019-11-22
EP3360890B1 (en) 2020-10-07
JP2024156706A (ja) 2024-11-06
ES2828050T3 (es) 2021-05-25
IL304084A (en) 2023-08-01
DK3262066T4 (da) 2023-08-21
JP2022141670A (ja) 2022-09-29
PL3262066T3 (pl) 2018-07-31
RU2018118954A (ru) 2019-12-02
WO2017072515A1 (en) 2017-05-04
CN116271104A (zh) 2023-06-23
CN108431030B (zh) 2023-10-20
BR112018008731A2 (en) 2018-11-06
RU2740038C2 (ru) 2020-12-30
JP7126944B2 (ja) 2022-08-29
CN116370655A (zh) 2023-07-04
EP3262066B2 (en) 2023-06-07
AU2022203675B2 (en) 2025-03-06
IL304084B2 (en) 2026-03-01
RU2018118954A3 (https=) 2020-03-13
AU2016344523B2 (en) 2022-03-17
AU2022203675A1 (en) 2022-06-16
IL284577B2 (en) 2023-12-01
IL284577A (en) 2021-08-31
EP3786176A1 (en) 2021-03-03
US20230277689A1 (en) 2023-09-07
GB201519086D0 (en) 2015-12-09
IL258806B (en) 2021-07-29
MX366343B (es) 2019-07-05
BR112018008731A8 (pt) 2020-03-31
EP3360890A1 (en) 2018-08-15
US20190255193A1 (en) 2019-08-22
PL3360890T3 (pl) 2020-12-28
CN108431030A (zh) 2018-08-21
AU2025200979A1 (en) 2025-03-06
DK3360890T3 (da) 2020-11-02
IL304084B1 (en) 2025-11-01
AU2016344523A1 (en) 2018-06-14
ZA201802462B (en) 2021-01-27
US20250319210A1 (en) 2025-10-16
IL284577B1 (en) 2023-08-01
EP3262066A1 (en) 2018-01-03
IL258806A (en) 2018-06-28
DK3262066T3 (en) 2018-05-22
CN117343960A (zh) 2024-01-05
PL3262066T5 (pl) 2023-10-30
ES2666204T3 (es) 2018-05-03
CA3002125A1 (en) 2017-05-04
SG11201802934WA (en) 2018-05-30
MX2018005145A (es) 2018-08-09
ES2666204T5 (es) 2023-11-03

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