CA3035605A1 - Gene therapy for patients with fanconi anemia - Google Patents

Gene therapy for patients with fanconi anemia Download PDF

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Publication number
CA3035605A1
CA3035605A1 CA3035605A CA3035605A CA3035605A1 CA 3035605 A1 CA3035605 A1 CA 3035605A1 CA 3035605 A CA3035605 A CA 3035605A CA 3035605 A CA3035605 A CA 3035605A CA 3035605 A1 CA3035605 A1 CA 3035605A1
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CA
Canada
Prior art keywords
cells
sequence
cell
fanca
vector
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.)
Pending
Application number
CA3035605A
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English (en)
French (fr)
Inventor
Juan A. BUEREN
Paula RIO
Susana NAVARRO
Julian SEVILLA
Jose Carlos SEGOVIA
Africa GONZALEZ
Jose Antonio CASADO
Guillermo GUENECHEA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
Original Assignee
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT
Centro de Investigacion Biomedica en Red CIBER
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus, Centro de Investigaciones Energeticas Medioambientales y Tecnologicas CIEMAT, Centro de Investigacion Biomedica en Red CIBER, Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz filed Critical Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Publication of CA3035605A1 publication Critical patent/CA3035605A1/en
Pending legal-status Critical Current

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1217—Phosphotransferases with a carboxyl group as acceptor (2.7.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
    • C12N2730/10143—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
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10171—Demonstrated in vivo effect
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15071—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (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)
CA3035605A 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia Pending CA3035605A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US62/385,185 2016-09-08
US201662412028P 2016-10-24 2016-10-24
US62/412,028 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Publications (1)

Publication Number Publication Date
CA3035605A1 true CA3035605A1 (en) 2018-03-15

Family

ID=61561668

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3035605A Pending CA3035605A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Country Status (11)

Country Link
US (1) US20190203225A1 (cg-RX-API-DMAC7.html)
EP (1) EP3510162A4 (cg-RX-API-DMAC7.html)
JP (3) JP7197466B2 (cg-RX-API-DMAC7.html)
KR (2) KR102672636B1 (cg-RX-API-DMAC7.html)
CN (1) CN110536966A (cg-RX-API-DMAC7.html)
AU (3) AU2017322511B2 (cg-RX-API-DMAC7.html)
CA (1) CA3035605A1 (cg-RX-API-DMAC7.html)
IL (2) IL265196B2 (cg-RX-API-DMAC7.html)
MX (1) MX2019002699A (cg-RX-API-DMAC7.html)
SG (1) SG11201901718VA (cg-RX-API-DMAC7.html)
WO (1) WO2018049273A1 (cg-RX-API-DMAC7.html)

Families Citing this family (14)

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MX382997B (es) 2016-04-20 2025-03-13 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Composiciones y metodos para mejorar la expresion genetica de pklr.
KR102726267B1 (ko) 2017-10-16 2024-11-11 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터
CA3093708A1 (en) * 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for treating fanconi anemia
WO2019210325A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
SG11202100022SA (en) * 2018-07-12 2021-01-28 Rocket Pharmaceuticals Ltd Gene therapy vectors for treatment of danon disease
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
CN110904102A (zh) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 一种用于重组蛋白质表达的启动子
WO2020169666A1 (en) * 2019-02-21 2020-08-27 F. Hoffmann-La Roche Ag Improved nucleic acid target enrichment and related methods
AU2020278046A1 (en) * 2019-05-23 2022-01-27 Spacecraft Seven, Llc Gene therapy vectors for infantile malignant osteopetrosis
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
EP4192487A4 (en) 2020-08-07 2024-10-02 Spacecraft Seven, LLC GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
WO2022232193A1 (en) * 2021-04-26 2022-11-03 Strm.Bio Incorporated Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
EP4499153A2 (en) * 2022-03-25 2025-02-05 ModernaTX, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2025137373A1 (en) * 2023-12-19 2025-06-26 Cellarity, Inc. Disease detection systems and methods

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US5952190A (en) * 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
GB0024550D0 (cg-RX-API-DMAC7.html) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
US8556882B2 (en) * 2009-04-30 2013-10-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
JP2014513727A (ja) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Ｃｘｃｒ４拮抗薬の使用
EP3808844A1 (en) * 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2014100073A2 (en) * 2012-12-21 2014-06-26 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
US9932588B2 (en) 2013-11-15 2018-04-03 Northwestern University Inhibition of oxidative stress in atrial fibrillation
US20170037431A1 (en) 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
EP3247808B1 (en) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2016183593A2 (en) * 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy

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Publication number Publication date
RU2019108981A (ru) 2020-10-08
JP2025043346A (ja) 2025-03-28
IL265196B1 (en) 2025-06-01
AU2017322511B2 (en) 2021-08-26
IL265196B2 (en) 2025-10-01
AU2017322511A1 (en) 2019-03-28
EP3510162A4 (en) 2020-02-19
SG11201901718VA (en) 2019-03-28
WO2018049273A1 (en) 2018-03-15
JP2022160505A (ja) 2022-10-19
RU2019108981A3 (cg-RX-API-DMAC7.html) 2020-12-24
KR102672636B1 (ko) 2024-06-10
AU2021273525A1 (en) 2021-12-16
BR112019004594A2 (pt) 2019-07-02
AU2021273525B2 (en) 2024-09-26
MX2019002699A (es) 2019-12-16
EP3510162A1 (en) 2019-07-17
KR20190062426A (ko) 2019-06-05
AU2024287211A1 (en) 2025-01-30
IL265196A (en) 2019-05-30
JP7197466B2 (ja) 2022-12-27
US20190203225A1 (en) 2019-07-04
CN110536966A (zh) 2019-12-03
JP2019533434A (ja) 2019-11-21
IL320371A (en) 2025-06-01
KR20240151726A (ko) 2024-10-18

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