MX381665B - Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos. - Google Patents

Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos.

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Publication number
MX381665B
MX381665B MX2018008651A MX2018008651A MX381665B MX 381665 B MX381665 B MX 381665B MX 2018008651 A MX2018008651 A MX 2018008651A MX 2018008651 A MX2018008651 A MX 2018008651A MX 381665 B MX381665 B MX 381665B
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MX
Mexico
Prior art keywords
osteoprotective
has2
lubricin
mammals
osteoarthritis
Prior art date
Application number
MX2018008651A
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English (en)
Other versions
MX2018008651A (es
Inventor
Figueiredo Monica Dias
Patricia Berthelette
Sirkka Rm Kyostio-Moore
Original Assignee
Genzyme Corp Star
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 Genzyme Corp Star filed Critical Genzyme Corp Star
Publication of MX2018008651A publication Critical patent/MX2018008651A/es
Publication of MX381665B publication Critical patent/MX381665B/es

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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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
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    • 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
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01212—Hyaluronan synthase (2.4.1.212)
    • 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
    • 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
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    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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

La presente invención se refiere a vectores virales recombinantes, composiciones farmacéuticas que comprenden dichos vectores recombinantes y métodos de prevención y tratamiento de la osteoartritis en mamíferos. En particular, esta divulgación provee vectores de virus adeno-asociados (AAV) capaces de expresar, en un huésped, proteínas bioactivas osteoprotectoras/condroprotectoras, que incluyen la hialurona sintasa 2 (HAS2) y lubricina (PRG4). Se proveen métodos de producción de estos AAV, así como métodos de tratamiento de la osteoartritis en las articulaciones de mamíferos, mediante la expresión genética a largo plazo de proteínas osteoprotectoras/condroprotectoras, que incluyen HAS2 y PRG4, en células sinoviales y condrocitos.
MX2018008651A 2016-01-13 2017-01-13 Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos. MX381665B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662278243P 2016-01-13 2016-01-13
PCT/US2017/013419 WO2017123934A1 (en) 2016-01-13 2017-01-13 Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals

Publications (2)

Publication Number Publication Date
MX2018008651A MX2018008651A (es) 2019-03-14
MX381665B true MX381665B (es) 2025-03-13

Family

ID=58054500

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018008651A MX381665B (es) 2016-01-13 2017-01-13 Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos.
MX2021004397A MX2021004397A (es) 2016-01-13 2018-07-12 Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos.

Family Applications After (1)

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MX2021004397A MX2021004397A (es) 2016-01-13 2018-07-12 Vectores aav recombinantes que expresan genes osteoprotectores, que incluyen has2 y lubricina, de utilidad en el tratamiento de la osteoartritis y afecciones articulares relacionadas en mamiferos.

Country Status (16)

Country Link
US (3) US20170198263A1 (es)
EP (2) EP3402530B1 (es)
JP (3) JP7058603B2 (es)
KR (1) KR20180095720A (es)
CN (2) CN108697813B (es)
AR (2) AR108683A1 (es)
AU (2) AU2017207917B2 (es)
CA (1) CA3011459A1 (es)
CY (1) CY1123760T1 (es)
DK (1) DK3402530T3 (es)
ES (1) ES2835031T3 (es)
HU (1) HUE051774T2 (es)
MX (2) MX381665B (es)
RU (1) RU2771490C2 (es)
WO (1) WO2017123934A1 (es)
ZA (1) ZA201804731B (es)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
LT3740222T (lt) 2018-01-17 2023-09-11 Meiragtx Uk Ii Limited Modifikuotas raav kapsidės baltymas, skirtas genų terapijai
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
CN109055426B (zh) * 2018-08-06 2021-06-25 智享生物(苏州)有限公司 一种在中华仓鼠卵巢细胞中表达生产类人润滑素的方法
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
EP3911355A4 (en) * 2019-01-15 2022-06-01 Cornell University RECOMBINANT LUBRICINS AND COMPOSITIONS AND METHODS OF USE THEREOF
EP3911369A4 (en) 2019-01-18 2022-11-16 Orthobio Therapeutics, Inc. GENE EDITING TO IMPROVE JOINT FUNCTION
WO2021086973A2 (en) * 2019-10-28 2021-05-06 University Of Iowa Research Foundation Formulation for delivery of lubricin gene
WO2021102250A1 (en) * 2019-11-21 2021-05-27 Luppino Francesco Growth factor restoration
KR102584136B1 (ko) * 2020-01-17 2023-10-04 주식회사 아바테라퓨틱스 조직 재생용 조성물
WO2022232129A1 (en) * 2021-04-26 2022-11-03 University Of Massachusetts Capsid variants and uses thereof
CN114457086B (zh) * 2022-03-02 2022-11-15 上海勉亦生物科技有限公司 白介素1受体拮抗蛋白的表达盒以及基于aav的基因递送系统
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
CN117721152B (zh) * 2023-12-29 2024-08-30 广州市第一人民医院（广州消化疾病中心、广州医科大学附属市一人民医院、华南理工大学附属第二医院） Ttp在制备骨关节炎药物中的应用

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6423514B1 (en) 1996-04-22 2002-07-23 Millennium Pharmaceuticals, Inc. Mammalian hyaluronan synthases, nucleic acids and uses thereof
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6743774B1 (en) * 1999-04-23 2004-06-01 Rhode Island Hospital Tribonectins
US6315992B1 (en) * 1999-06-30 2001-11-13 Tissuegene Co. Generating cartilage in a mammal using fibroblasts transfected with a vector encoding TGF-β-1
US7338655B1 (en) * 1999-06-30 2008-03-04 Tissuegene, Inc. Gene therapy using TGF-β
JP4827353B2 (ja) 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
JP2003164289A (ja) 2001-11-30 2003-06-10 Seikagaku Kogyo Co Ltd 骨組織導入用遺伝子
EP1593668A4 (en) 2003-02-14 2007-07-11 Wako Pure Chem Ind Ltd METHOD FOR PRODUCING A HYDROXYLAMINE COMPOUND USING A PLATINATE CATALYST IMMOBILIZED ON AN ION EXCHANGE RESIN
US20060240037A1 (en) 2003-06-04 2006-10-26 Edward Fey Methods and compositions for the treatment and prevention of degenerative joint disorders
AU2004264970B2 (en) 2003-08-14 2010-02-11 Wyeth Recombinant lubricin molecules and uses thereof
NZ545656A (en) * 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
PL1664315T3 (pl) 2003-09-01 2012-09-28 Acad Medisch Ct Wektory aav do terapii genowej in vivo reumatoidalnego zapalenia stawów
WO2005102363A2 (en) 2004-04-20 2005-11-03 Mucosal Therapeutics Llc Methods of promoting cartilage healing or cartilage integration
EP1827478A4 (en) 2004-12-03 2009-08-05 Mucosal Therapeutics Llc METHODS OF TREATING JOINED OR SICK JOINTS
US7771995B2 (en) 2005-11-14 2010-08-10 Merial Limited Plasmid encoding human BMP-7
US8298818B2 (en) * 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
US20080187576A1 (en) 2006-06-16 2008-08-07 University Of Florida Research Foundation, Inc. Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
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
US7846428B2 (en) 2007-10-05 2010-12-07 Merial Limited Articular cartilage gene therapy with recombinant vector encoding BMP-7
KR101923152B1 (ko) * 2007-12-04 2018-11-28 프로테오바이오엑티브스 피티와이 엘티디 전구 세포의 보호 및 그의 분화 조절
EP3043179B1 (en) 2009-04-01 2020-05-06 Galapagos N.V. Methods and means for treatment of osteoarthritis
WO2011005493A2 (en) 2009-06-22 2011-01-13 Mayo Foundation For Medical Education And Research Methods and materials for tissue repair
ES2628889T3 (es) * 2010-02-05 2017-08-04 The University Of North Carolina At Chapel Hill Composiciones y métodos para la transducción mejorada del parvovirus
PH12013501636A1 (en) * 2011-02-08 2013-10-14 Abbvie Inc Treatment of osteoarthritis and pain
US20130116186A1 (en) 2011-10-04 2013-05-09 Rhode Island Hospital, A Lifespan Partner Lubricin injections to maintain cartilage health
DK2623604T3 (da) * 2012-02-02 2015-06-01 Baylor College Medicine Adenovirusbaseret biologisk afgivelses- og ekspressionssystem til anvendelse ved behandling af osteoartrose
AU2013243952A1 (en) * 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
EP2948553B1 (en) 2013-01-25 2020-04-01 Baylor College Of Medicine A helper-dependent adenoviral gene therapy delivery and expression system
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
WO2014205237A2 (en) * 2013-06-19 2014-12-24 University Of Rochester Compositions and methods for use of high molecular weight hyaluronic acid for cancer therapy
AU2014317861B2 (en) * 2013-09-09 2019-11-28 Figene, Llc Gene therapy for the regeneration of chondrocytes or cartilage type cells
AU2015330092A1 (en) * 2014-10-06 2017-04-13 Arthrogen B.V. AAV-based gene therapy

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Publication number Publication date
ZA201804731B (en) 2019-05-29
AR130990A2 (es) 2025-02-05
KR20180095720A (ko) 2018-08-27
US20170198263A1 (en) 2017-07-13
DK3402530T3 (da) 2020-11-23
US11905531B2 (en) 2024-02-20
RU2018129159A (ru) 2020-02-13
AU2024203920A1 (en) 2024-07-11
JP2022058602A (ja) 2022-04-12
ES2835031T3 (es) 2021-06-21
RU2771490C2 (ru) 2022-05-05
CA3011459A1 (en) 2017-07-20
JP2019506859A (ja) 2019-03-14
AU2017207917A1 (en) 2018-08-16
US20200231940A1 (en) 2020-07-23
MX2021004397A (es) 2021-06-08
AU2017207917B2 (en) 2024-03-07
MX2018008651A (es) 2019-03-14
JP7058603B2 (ja) 2022-04-22
JP2025019058A (ja) 2025-02-06
CN108697813B (zh) 2024-01-16
CN108697813A (zh) 2018-10-23
AR108683A1 (es) 2018-09-19
EP3769788A1 (en) 2021-01-27
CY1123760T1 (el) 2022-03-24
US20240254454A1 (en) 2024-08-01
WO2017123934A1 (en) 2017-07-20
RU2018129159A3 (es) 2020-03-27
EP3402530B1 (en) 2020-08-19
CN118048321A (zh) 2024-05-17
HUE051774T2 (hu) 2021-03-29
EP3402530A1 (en) 2018-11-21
JP7629584B2 (ja) 2025-02-14

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