MX2019013836A - Vectores virales que codifican variantes del factor de coagulacion ix (fix) recombinantes con mayor expresion para la terapia genica de hemofilia b. - Google Patents
Vectores virales que codifican variantes del factor de coagulacion ix (fix) recombinantes con mayor expresion para la terapia genica de hemofilia b.Info
- Publication number
- MX2019013836A MX2019013836A MX2019013836A MX2019013836A MX2019013836A MX 2019013836 A MX2019013836 A MX 2019013836A MX 2019013836 A MX2019013836 A MX 2019013836A MX 2019013836 A MX2019013836 A MX 2019013836A MX 2019013836 A MX2019013836 A MX 2019013836A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- hemophilia
- viral vectors
- increased expression
- vectors encoding
- Prior art date
Links
Classifications
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- 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
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
- A61K48/0066—Manipulation 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
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- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- 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
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- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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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
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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
- C12N15/86—Viral vectors
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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
- 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
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Hematology (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
La presente descripción proporciona, entre otros aspectos, polinucleótidos modificados con codones que codifican variantes del Factor IX para expresión en células de mamífero. En algunas modalidades, la descripción también proporciona vectores de terapia génica de mamífero y métodos para tratar la hemofilia B.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762509616P | 2017-05-22 | 2017-05-22 | |
PCT/US2018/033866 WO2018217731A1 (en) | 2017-05-22 | 2018-05-22 | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2019013836A true MX2019013836A (es) | 2020-01-30 |
Family
ID=62621009
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019013836A MX2019013836A (es) | 2017-05-22 | 2018-05-22 | Vectores virales que codifican variantes del factor de coagulacion ix (fix) recombinantes con mayor expresion para la terapia genica de hemofilia b. |
Country Status (16)
Country | Link |
---|---|
US (2) | US10842853B2 (es) |
EP (1) | EP3630974A1 (es) |
JP (1) | JP6961171B2 (es) |
KR (2) | KR102386890B1 (es) |
AR (1) | AR112057A1 (es) |
AU (1) | AU2018272831A1 (es) |
BR (1) | BR112019024256A2 (es) |
CA (1) | CA3064730A1 (es) |
CL (2) | CL2019003395A1 (es) |
CO (1) | CO2019012885A2 (es) |
IL (2) | IL270567B (es) |
MX (1) | MX2019013836A (es) |
NZ (1) | NZ759034A (es) |
TW (1) | TWI753168B (es) |
WO (1) | WO2018217731A1 (es) |
ZA (1) | ZA201907716B (es) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
US11596671B2 (en) * | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
BR112021017062A2 (pt) * | 2019-03-19 | 2021-11-16 | CSL Behring Lengnau AG | Variantes do fator ix e usos das mesmas em terapia |
US20200405883A1 (en) | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
TW202246505A (zh) * | 2021-03-05 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | 編碼凝血因子ix蛋白的密碼子優化的核酸及其用途 |
CN114277057B (zh) * | 2021-07-09 | 2023-10-13 | 上海天泽云泰生物医药有限公司 | 用于治疗或预防b型血友病的重组腺相关病毒载体和方法 |
AU2022379633A1 (en) * | 2021-10-27 | 2024-04-11 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
Family Cites Families (28)
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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 |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
WO1994029471A1 (en) | 1993-06-10 | 1994-12-22 | Genetic Therapy, Inc. | Adenoviral vectors for treatment of hemophilia |
US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
CN1172435A (zh) | 1994-09-23 | 1998-02-04 | 综合医院公司 | 使用非哺乳动物dna病毒在哺乳动物细胞中表达外源基因 |
US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
US5858351A (en) | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
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 |
US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
US6093392A (en) | 1997-03-14 | 2000-07-25 | Childrens Hospital Of Phildelphia | Methods and compositions for use in gene therapy for treatment of hemophilia |
US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
AU5470900A (en) | 1999-06-08 | 2000-12-28 | Children's Hospital Of Philadelphia, The | Methods for preventing formation of inhibitory antibodies in the setting of genetherapy |
CN1148228C (zh) | 2000-08-30 | 2004-05-05 | 夏家辉 | 治疗血友病b的基因药物及其制备方法 |
EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
EP2438931B1 (en) * | 2004-09-22 | 2013-11-13 | St. Jude Children's Research Hospital | Improved expression of factor IX in gene therapy vectors |
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 |
WO2009130208A1 (en) | 2008-04-22 | 2009-10-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
DK3581650T3 (da) | 2008-09-15 | 2023-03-13 | Uniqure Biopharma B V | Faktor ix polypeptidmutant, anvendelse deraf og en fremgangsmåde til fremstilling deraf |
TWI595004B (zh) | 2010-11-03 | 2017-08-11 | 介控生化科技公司 | 經修飾之第九因子多胜肽及其用途 |
CA2856137A1 (en) | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
US20150111955A1 (en) | 2012-02-17 | 2015-04-23 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
WO2014063753A1 (en) | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof |
CA2888931C (en) * | 2012-10-26 | 2023-09-05 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
AU2016232146B2 (en) | 2015-03-17 | 2021-11-04 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
IL256517B2 (en) | 2015-06-23 | 2024-06-01 | Childrens Hospital Philadelphia | Modified factor IX, and preparations, methods and uses for gene transfer to cells, organs and tissues |
-
2018
- 2018-05-22 BR BR112019024256A patent/BR112019024256A2/pt unknown
- 2018-05-22 KR KR1020197037828A patent/KR102386890B1/ko active IP Right Grant
- 2018-05-22 WO PCT/US2018/033866 patent/WO2018217731A1/en unknown
- 2018-05-22 NZ NZ759034A patent/NZ759034A/en unknown
- 2018-05-22 US US15/986,350 patent/US10842853B2/en active Active
- 2018-05-22 CA CA3064730A patent/CA3064730A1/en active Pending
- 2018-05-22 JP JP2019564430A patent/JP6961171B2/ja active Active
- 2018-05-22 AR ARP180101356A patent/AR112057A1/es unknown
- 2018-05-22 TW TW107117388A patent/TWI753168B/zh active
- 2018-05-22 MX MX2019013836A patent/MX2019013836A/es unknown
- 2018-05-22 KR KR1020227012028A patent/KR102551733B1/ko active IP Right Grant
- 2018-05-22 AU AU2018272831A patent/AU2018272831A1/en active Pending
- 2018-05-22 EP EP18731636.9A patent/EP3630974A1/en active Pending
-
2019
- 2019-11-11 IL IL270567A patent/IL270567B/en active IP Right Grant
- 2019-11-18 CO CONC2019/0012885A patent/CO2019012885A2/es unknown
- 2019-11-21 ZA ZA2019/07716A patent/ZA201907716B/en unknown
- 2019-11-21 CL CL2019003395A patent/CL2019003395A1/es unknown
-
2020
- 2020-09-23 CL CL2020002445A patent/CL2020002445A1/es unknown
- 2020-10-20 US US17/075,556 patent/US20210128700A1/en active Pending
-
2021
- 2021-02-04 IL IL280637A patent/IL280637A/en unknown
Also Published As
Publication number | Publication date |
---|---|
IL280637A (en) | 2021-03-25 |
US10842853B2 (en) | 2020-11-24 |
CL2020002445A1 (es) | 2020-12-28 |
US20180339026A1 (en) | 2018-11-29 |
RU2019142702A3 (es) | 2021-10-12 |
EP3630974A1 (en) | 2020-04-08 |
KR102551733B1 (ko) | 2023-07-06 |
ZA201907716B (en) | 2022-06-29 |
WO2018217731A1 (en) | 2018-11-29 |
TW201900184A (zh) | 2019-01-01 |
CO2019012885A2 (es) | 2020-01-17 |
RU2019142702A (ru) | 2021-06-24 |
JP2020520662A (ja) | 2020-07-16 |
IL270567B (en) | 2021-03-25 |
AR112057A1 (es) | 2019-09-18 |
NZ759034A (en) | 2023-07-28 |
US20210128700A1 (en) | 2021-05-06 |
KR102386890B1 (ko) | 2022-04-15 |
CA3064730A1 (en) | 2018-11-29 |
KR20200010443A (ko) | 2020-01-30 |
AU2018272831A1 (en) | 2019-11-28 |
CL2019003395A1 (es) | 2020-04-17 |
CN110945127A (zh) | 2020-03-31 |
TWI753168B (zh) | 2022-01-21 |
KR20220050238A (ko) | 2022-04-22 |
BR112019024256A2 (pt) | 2020-06-09 |
JP6961171B2 (ja) | 2021-11-05 |
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