CA2849241C - Construct - Google Patents

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Publication number
CA2849241C
CA2849241C CA2849241A CA2849241A CA2849241C CA 2849241 C CA2849241 C CA 2849241C CA 2849241 A CA2849241 A CA 2849241A CA 2849241 A CA2849241 A CA 2849241A CA 2849241 C CA2849241 C CA 2849241C
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CA
Canada
Prior art keywords
vector
aadc
dopamine
viral vector
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2849241A
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English (en)
French (fr)
Other versions
CA2849241A1 (en
Inventor
Kyriacos Mitrophanous
Scott Ralph
Hannah Stewart
Alan Kingsman
Original Assignee
Oxford Biomedica UK Ltd
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 Oxford Biomedica UK Ltd filed Critical Oxford Biomedica UK Ltd
Priority to CA3052407A priority Critical patent/CA3052407C/en
Publication of CA2849241A1 publication Critical patent/CA2849241A1/en
Application granted granted Critical
Publication of CA2849241C publication Critical patent/CA2849241C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • C12N15/867—Retroviral 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P13/00—Preparation of nitrogen-containing organic compounds
    • C12P13/001—Amines; Imines
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2849241A 2011-10-28 2012-10-26 Construct Active CA2849241C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3052407A CA3052407C (en) 2011-10-28 2012-10-26 Construct

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161552581P 2011-10-28 2011-10-28
GB1118636.8 2011-10-28
US61/552,581 2011-10-28
GBGB1118636.8A GB201118636D0 (en) 2011-10-28 2011-10-28 Nucleotide sequence
PCT/GB2012/052668 WO2013061076A1 (en) 2011-10-28 2012-10-26 Construct

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3052407A Division CA3052407C (en) 2011-10-28 2012-10-26 Construct

Publications (2)

Publication Number Publication Date
CA2849241A1 CA2849241A1 (en) 2013-05-02
CA2849241C true CA2849241C (en) 2019-12-31

Family

ID=45373567

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2849241A Active CA2849241C (en) 2011-10-28 2012-10-26 Construct
CA3052407A Active CA3052407C (en) 2011-10-28 2012-10-26 Construct

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3052407A Active CA3052407C (en) 2011-10-28 2012-10-26 Construct

Country Status (11)

Country Link
US (1) US11279954B2 (enExample)
EP (2) EP3219801B1 (enExample)
JP (1) JP6296987B2 (enExample)
CN (2) CN104011213B (enExample)
AU (1) AU2012328166B2 (enExample)
CA (2) CA2849241C (enExample)
DK (2) DK2771471T3 (enExample)
ES (2) ES2857003T3 (enExample)
GB (1) GB201118636D0 (enExample)
PL (2) PL2771471T3 (enExample)
WO (1) WO2013061076A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
WO2017021359A1 (en) * 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
GB2562774A (en) * 2017-05-25 2018-11-28 Mcdonald Michael Genetic construct
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技（杭州）有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN115835887A (zh) * 2019-03-10 2023-03-21 牛津生物医学（英国）有限公司 用于治疗帕金森病的基因治疗组合物和方法
CN109971729B (zh) * 2019-04-19 2021-07-16 上海信致医药科技有限公司 一种酶组合物
GB2587319A (en) 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
CN116555351B (zh) * 2020-03-13 2024-10-18 康霖生物科技（杭州）有限公司 一种核酸构建体
GB2601752A (en) 2020-12-08 2022-06-15 Maavrx Ltd Expression vector
CN118715325A (zh) 2021-12-17 2024-09-27 帝国理工创新有限公司 Rna构建体
CN114317610B (zh) * 2022-01-27 2024-07-02 上海本导基因技术有限公司 一种适用于帕金森疾病基因治疗的慢病毒载体
JP2025516136A (ja) * 2022-04-19 2025-05-27 シャンハイ ヴァイタルゲン バイオファーマ カンパニー リミテッド 神経変性疾患の治療のための組み換えａａｖベクター
CN117106825B (zh) * 2023-08-28 2024-11-01 康霖生物科技（杭州）有限公司 用于治疗帕金森病的基因治疗载体及其用途
CN118389675B (zh) * 2024-05-27 2025-06-27 南方医科大学珠江医院 芳香族氨基酸脱羧酶作为标志物在肝性脑病的检测产品中的应用

Family Cites Families (27)

* 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
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
DE4006630A1 (de) 1990-03-03 1991-09-12 Behringwerke Ag Humane monoklonale antikoerper gegen tollwutviren, ihre herstellung und verwendung
US5512421A (en) 1991-02-19 1996-04-30 The Regents Of The University Of California Generation, concentration and efficient transfer of VSV-G pseudotyped retroviral vectors
AU671593B2 (en) 1991-06-21 1996-09-05 Uop Llc Method for increasing rainout from autorefrigerated vaporous cloud containing hydrofluoric acid
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
AU1073797A (en) * 1995-11-14 1997-06-05 Somatix Therapy Corporation Joint expression of gtp cyclohydrolase and tyrosine hydroxylase
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
GB9622500D0 (en) * 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
DE69941049D1 (de) 1998-05-22 2009-08-13 Oxford Biomedica Ltd Retrovirales verabreichungssystem
GB9904905D0 (en) 1999-03-03 1999-04-28 Oxford Biomedica Ltd Cells
WO2001004433A1 (es) 1999-07-13 2001-01-18 Pellicer Carlos F Panel para la construccion e instalacion para su fabricacion
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
GB0024550D0 (enExample) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
DK2348119T3 (en) 2002-02-01 2017-08-21 Oxford Biomedica (Uk) Ltd Polycistronic lentiviral vector
SE0302509D0 (sv) 2003-09-19 2003-09-19 Amersham Biosciences Ab Matrix for separation of polyethers and method of separation
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
GB0526210D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2009153563A1 (en) 2008-06-18 2009-12-23 Oxford Biomedica (Uk) Limited Virus purification
JP5559185B2 (ja) * 2008-11-11 2014-07-23 オックスフォード バイオメディカ（ユーケー）リミテッド 方法
CN102946907A (zh) * 2010-05-28 2013-02-27 牛津生物医学（英国）有限公司 慢病毒载体向脑的递送

Also Published As

Publication number Publication date
CN104011213A (zh) 2014-08-27
AU2012328166A1 (en) 2014-06-12
US20200040361A1 (en) 2020-02-06
GB201118636D0 (en) 2011-12-07
DK2771471T3 (en) 2017-08-21
JP2014532402A (ja) 2014-12-08
EP3219801B1 (en) 2020-12-16
CN107523581A (zh) 2017-12-29
AU2012328166B2 (en) 2018-03-08
WO2013061076A1 (en) 2013-05-02
EP3219801A1 (en) 2017-09-20
ES2628419T3 (es) 2017-08-02
JP6296987B2 (ja) 2018-03-20
EP2771471B1 (en) 2017-05-03
CA3052407A1 (en) 2013-05-02
EP2771471A1 (en) 2014-09-03
US11279954B2 (en) 2022-03-22
CA3052407C (en) 2020-03-31
CA2849241A1 (en) 2013-05-02
PL2771471T3 (pl) 2017-10-31
CN104011213B (zh) 2017-10-03
PL3219801T3 (pl) 2021-06-14
CN107523581B (zh) 2021-08-17
DK3219801T3 (da) 2021-02-22
ES2857003T3 (es) 2021-09-28

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