AU2014287005C1 - AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies - Google Patents

AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies Download PDF

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AU2014287005C1
AU2014287005C1 AU2014287005A AU2014287005A AU2014287005C1 AU 2014287005 C1 AU2014287005 C1 AU 2014287005C1 AU 2014287005 A AU2014287005 A AU 2014287005A AU 2014287005 A AU2014287005 A AU 2014287005A AU 2014287005 C1 AU2014287005 C1 AU 2014287005C1
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aav
cells
vector
sequence
biological sample
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AU2014287005A1 (en
AU2014287005B2 (en
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Yifeng Chen
Katherine A. High
Federico Mingozzi
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Childrens Hospital of Philadelphia CHOP
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    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6854—Immunoglobulins
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • C12Q1/701—Specific hybridization probes
    • 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
    • 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
    • C12N2750/14131—Uses of virus other than therapeutic or vaccine, e.g. disinfectant
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
    • G01N2333/01—DNA viruses
    • G01N2333/075—Adenoviridae
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/902—Oxidoreductases (1.)
    • G01N2333/90241—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2469/00—Immunoassays for the detection of microorganisms
    • G01N2469/20—Detection of antibodies in sample from host which are directed against antigens from microorganisms

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Analytical Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
AU2014287005A 2013-07-12 2014-07-11 AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies Active AU2014287005C1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361845841P 2013-07-12 2013-07-12
US61/845,841 2013-07-12
PCT/US2014/046428 WO2015006743A1 (en) 2013-07-12 2014-07-11 Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies

Publications (3)

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AU2014287005A1 AU2014287005A1 (en) 2016-01-28
AU2014287005B2 AU2014287005B2 (en) 2020-12-17
AU2014287005C1 true AU2014287005C1 (en) 2021-06-03

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US (3) US11408899B2 (https=)
EP (2) EP4039813A1 (https=)
JP (4) JP2016525347A (https=)
KR (1) KR102445330B1 (https=)
CN (2) CN105518145A (https=)
AU (1) AU2014287005C1 (https=)
CA (1) CA2918038C (https=)
HK (1) HK1222673A1 (https=)
IL (1) IL243438B (https=)
MX (2) MX2016000345A (https=)
RU (1) RU2705249C2 (https=)
WO (1) WO2015006743A1 (https=)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016164920A1 (en) * 2015-04-09 2016-10-13 Cornell University Gene therapy to prevent reactions to allergens
WO2016210170A1 (en) 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
WO2017096162A1 (en) * 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US20210228738A1 (en) * 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
CN107828821A (zh) * 2017-11-15 2018-03-23 四川大学 一种腺相关病毒感染敏感的细胞系的构建方法及腺相关病毒中和抗体含量测定的检测方法
CA3086046C (en) * 2017-12-29 2023-02-21 Helixmith Co., Ltd. Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto
CN111902164A (zh) 2018-02-01 2020-11-06 同源药物公司 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
EP3775173A1 (en) * 2018-04-05 2021-02-17 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
EP3796980A4 (en) * 2018-05-22 2022-03-23 The Brigham & Women's Hospital, Inc. NEW THERAPY FOR ALZHEIMER'S DISEASE
CN110846392A (zh) * 2018-08-20 2020-02-28 武汉纽福斯生物科技有限公司 一种重组腺相关病毒或含其的试剂盒及其应用
MX2021005727A (es) * 2018-11-16 2021-09-10 Spark Therapeutics Inc Ensayo in vitro para detectar potenciadores e inhibidores de la transducción de vectores virales adenoasociados (aav) y/o para detectar o cuantificar anticuerpos de unión anti-aav.
EP3918308A4 (en) * 2019-01-31 2023-09-27 Precision Bioservices, Inc. METHOD FOR DETECTING AND CHARACTERIZING ANTIVIRAL VECTOR ANTIBODIES
AU2020263757B2 (en) 2019-04-24 2025-08-28 Takara Bio Inc. AAV mutant having brain-targeting property
WO2020242892A1 (en) 2019-05-24 2020-12-03 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
IL293285A (en) 2019-11-29 2022-07-01 Paros Bio Inc Gene therapy for neurodegenerative disorders
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
KR20220133969A (ko) 2020-01-28 2022-10-05 프리라인 테라퓨틱스 리미티드 바이러스 벡터에 대한 중화 항체 역가를 결정하기 위한 개선된 분석법
WO2021209275A1 (en) * 2020-04-12 2021-10-21 Novoscope Ip Limited Particles, dna & rna
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
PL4211231T3 (pl) * 2020-09-10 2024-11-04 Genethon Kapsyd AAV modyfikowany peptydem
CN112143712B (zh) * 2020-09-30 2022-11-01 中国科学院深圳先进技术研究院 一种重组腺相关病毒及其制备方法和在抗体检测的应用
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
CN114277090A (zh) * 2021-12-17 2022-04-05 宁波熙宁检测技术有限公司 Aav8中和抗体检测方法和检测试剂盒
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023207918A1 (en) * 2022-04-25 2023-11-02 Beijing Hanmoluojie Technology Co., Ltd. Aav capsid 3d molecular surface feature mapping
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
CN119562959A (zh) * 2022-05-13 2025-03-04 费城儿童医院 用于靶向脑微脉管系统的腺相关病毒载体
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
CN115047188A (zh) * 2022-06-10 2022-09-13 上海鼎新基因科技有限公司 一种抗腺相关病毒中和抗体的检测方法
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
US20250352580A1 (en) * 2022-11-04 2025-11-20 The Children's Hospital Of Philadelphia Compositions and methods for b cell directed immunotherapies for anti-aav neutralizing alloantibodies
CN117285620B (zh) * 2023-11-27 2024-02-13 恺佧生物科技(上海)有限公司 抗aav9抗体及aav9滴度测定elisa试剂盒
WO2025119252A1 (en) * 2023-12-06 2025-06-12 Immvira Biopharmaceuticals Co., Limited Methods for detecting an anti-hsv antibody in a sample
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013078400A1 (en) * 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
DE69841982D1 (de) * 1997-03-14 2010-12-16 Philadelphia Children Hospital Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie
JP4025400B2 (ja) * 1997-04-16 2007-12-19 財団法人化学及血清療法研究所 新規免疫制御分子及びその製造方法
EP1082413B1 (en) * 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
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
DK1035133T3 (da) * 1999-02-17 2005-05-02 Pfizer Prod Inc Fusionsproteiner, der omfatter bærere, som kan inducere et dobbelt immunrespons
EP1222299A1 (en) * 1999-10-01 2002-07-17 Genovo, Incorporated Production of recombinant aav using adenovirus comprising aav rep/cap genes
US7241447B1 (en) * 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
NZ522840A (en) * 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
DE50304957D1 (de) * 2002-04-15 2006-10-19 Universitaetsklinikum Hamburg Rekombinante virale vektoren zur tetracyclinregulierbaren genexpression
EP1369491A1 (en) 2002-06-05 2003-12-10 Aladar A. Szalay Light emitting microorganisms and cells for diagnosis and therapy of diseases associated with wounded or inflamed tissue
AU2003274397A1 (en) * 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
KR20050010916A (ko) 2002-06-13 2005-01-28 와이어쓰 홀딩스 코포레이션 염증 유전자 활성 및 콜레스테롤 생합성의 억제제
JP2012503771A (ja) * 2008-09-25 2012-02-09 デューク ユニバーシティー 抗体および抗体−ｈｉｖビリオン複合体を検出する方法
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
CN103167896B (zh) * 2010-08-20 2015-12-16 耐克创新有限合伙公司 高尔夫球杆和高尔夫球杆头
JP4916596B1 (ja) 2010-10-01 2012-04-11 パナソニック株式会社 脂質二重膜の表側から裏側にカリウムイオンを輸送する方法
US8202687B2 (en) 2010-10-01 2012-06-19 Panasonic Corporation Method for transporting potassium ions from front side to back side of lipid bilayer membrane
SG10202103336SA (en) 2010-11-02 2021-04-29 Promega Corp Novel coelenterazine substrates and methods of use
ES2685611T3 (es) * 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
CN103014064A (zh) 2011-09-22 2013-04-03 北京五加和分子医学研究所有限公司 Itr同向排列的aav载体构建及其应用

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013078400A1 (en) * 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Calcedo R et al, Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses", Journal of Infectious Diseases, 2009, 199(3):381-390. doi: 10.1086/595830. *
Kay MA et al, "Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector", Nature Genetics, 2000, 24(3):257-261, doi:10.1038/73464 *
Manno CS et al, "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response", Nature Medicine, 2006, 12(3):342-347, doi:10.1038/nm1358 *
Sen D et al, "Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo", Human Gene Therapy Methods, 2013, 24(2):104-116, doi: 10.1089/hgtb.2012.195. *

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AU2014287005A1 (en) 2016-01-28
EP4039813A1 (en) 2022-08-10
EP3019620A1 (en) 2016-05-18
RU2016104614A3 (https=) 2018-03-05
KR102445330B1 (ko) 2022-09-19
US20160123990A1 (en) 2016-05-05
JP2019162109A (ja) 2019-09-26
JP2022037027A (ja) 2022-03-08
US20260043810A1 (en) 2026-02-12
KR20160034332A (ko) 2016-03-29
CA2918038C (en) 2023-03-28
US20220334126A1 (en) 2022-10-20
JP2016525347A (ja) 2016-08-25
CA2918038A1 (en) 2015-01-15
US11408899B2 (en) 2022-08-09
IL243438B (en) 2020-02-27
RU2705249C2 (ru) 2019-11-06
WO2015006743A1 (en) 2015-01-15
HK1222673A1 (zh) 2017-07-07
MX2021000675A (es) 2021-03-25
AU2014287005B2 (en) 2020-12-17
RU2016104614A (ru) 2017-08-18
JP2024026253A (ja) 2024-02-28
MX2016000345A (es) 2016-06-21
EP3019620A4 (en) 2017-01-18
CN105518145A (zh) 2016-04-20
CN116004721A (zh) 2023-04-25
IL243438A0 (en) 2016-03-31

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