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MX2018005145A - Terapia genetica. - Google Patents

Terapia genetica.

Info

Publication number
MX2018005145A
MX2018005145A MX2018005145A MX2018005145A MX2018005145A MX 2018005145 A MX2018005145 A MX 2018005145A MX 2018005145 A MX2018005145 A MX 2018005145A MX 2018005145 A MX2018005145 A MX 2018005145A MX 2018005145 A MX2018005145 A MX 2018005145A
Authority
MX
Mexico
Prior art keywords
genetic therapy
factor
vector
aav
adeno
Prior art date
Application number
MX2018005145A
Other languages
English (en)
Other versions
MX366343B (es
Inventor
GROENDAHL Christian
Funnell Tim
Hollowood Chris
Original Assignee
Syncona Investment Man Limited
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
Family has litigation
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=MX2018005145(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Syncona Investment Man Limited filed Critical Syncona Investment Man Limited
Publication of MX2018005145A publication Critical patent/MX2018005145A/es
Publication of MX366343B publication Critical patent/MX366343B/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • A61K48/0075Medicinal 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

Landscapes

  • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hematology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)

Abstract

Un vector viral adeno-asociado (AAV), el cual comprende una secuencia de nucleótidos que codifica el Factor I o el Factor H, o un fragmento o derivado de los mismos. El vector es útil para el tratamiento o la prevención de un trastorno del ojo mediado por el complemento.
MX2018005145A 2015-10-28 2016-10-27 Terapia genetica. MX366343B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
MX2018005145A true MX2018005145A (es) 2018-08-09
MX366343B MX366343B (es) 2019-07-05

Family

ID=55130363

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018005145A MX366343B (es) 2015-10-28 2016-10-27 Terapia genetica.

Country Status (18)

Country Link
US (3) US20190255193A1 (es)
EP (3) EP3360890B1 (es)
JP (3) JP7126944B2 (es)
CN (4) CN117343960A (es)
AU (3) AU2016344523B2 (es)
CA (1) CA3002125A1 (es)
DK (2) DK3262066T4 (es)
ES (2) ES2666204T5 (es)
GB (1) GB201519086D0 (es)
HK (1) HK1248723B (es)
IL (3) IL304084B1 (es)
MX (1) MX366343B (es)
NO (1) NO3262066T3 (es)
PL (2) PL3262066T5 (es)
RU (1) RU2740038C2 (es)
SG (1) SG11201802934WA (es)
WO (1) WO2017072515A1 (es)
ZA (1) ZA201802462B (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL258024B2 (en) 2015-09-24 2023-03-01 Univ Pennsylvania A preparation and method for the treatment of a complement-mediated disease
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
EP3452103A1 (en) 2016-04-15 2019-03-13 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
CN111630170B (zh) * 2017-07-31 2025-03-07 映像生物有限公司 眼部疾病的细胞模型及用于眼部疾病的疗法
EP3697920A4 (en) * 2017-10-20 2022-03-02 Gemini Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
CA3115782A1 (en) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Kir 7.1 gene therapy vectors and methods of using the same
EP3870711A1 (en) * 2018-10-23 2021-09-01 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
JP2022521851A (ja) * 2019-04-03 2022-04-12 レジェンクスバイオ インコーポレーテッド 眼の病態に対する遺伝子療法
BR112022003508A2 (pt) * 2019-09-09 2022-06-28 Univ Pittsburgh Commonwealth Sys Higher Education Métodos para restaurar a função lisossômica de células epiteliais de pigmento da retina por ativação de tfeb
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
AU2021340972A1 (en) * 2020-09-09 2023-04-13 Homology Medicines, Inc. Vectorized antibodies and uses thereof
EP4238567A4 (en) 2020-10-30 2024-09-04 Keio University NEW TREATMENT AND PREVENTION OF DISEASES RELATED TO SARCOPENIA
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 (es) 2022-01-25 2023-08-03

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
JP5623685B2 (ja) * 2003-06-10 2014-11-12 エヌエスゲーネ・アクティーゼルスカブ ニューブラスチンの改良された分泌
WO2006088950A2 (en) 2005-02-14 2006-08-24 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
AU2006304804B2 (en) 2005-10-21 2011-06-02 Vertex Pharmaceuticals Incorporated Modified proteases that inhibit complement activation
AU2007261315B2 (en) 2006-06-21 2012-10-25 Musc Foundation For Research Development Targeting complement factor H for treatment of diseases
KR20090122465A (ko) 2007-03-01 2009-11-30 어드벤스드 비젼 테라피스, 인코포레이티드 염증성 질환의 치료
CN101657097A (zh) * 2007-03-01 2010-02-24 先进视觉疗法公司 以炎症为特征的疾病的治疗
PL2191001T4 (pl) 2007-04-09 2017-01-31 University Of Florida Research Foundation, Inc. Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania
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
TWI698240B (zh) * 2012-05-15 2020-07-11 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)
JP6563815B2 (ja) * 2013-01-23 2019-08-21 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 天然抗体に由来する構築物を標的化すること及びその使用
US20140234275A1 (en) * 2013-02-15 2014-08-21 Jason Williams Method for treating als via the increased production of factor h
CN105682674A (zh) * 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
EP3013946B8 (en) * 2013-06-28 2019-04-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for expressing a polynucleotide of interest in the retina of a subject
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
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

Also Published As

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

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