DK2282764T3 - Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf - Google Patents
Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser herafInfo
- Publication number
- DK2282764T3 DK2282764T3 DK09735371.8T DK09735371T DK2282764T3 DK 2282764 T3 DK2282764 T3 DK 2282764T3 DK 09735371 T DK09735371 T DK 09735371T DK 2282764 T3 DK2282764 T3 DK 2282764T3
- Authority
- DK
- Denmark
- Prior art keywords
- applications
- methods
- nucleic acid
- regulatory elements
- specific nucleic
- 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
- 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
- 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
- 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
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
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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
- C12N15/52—Genes encoding for enzymes or proenzymes
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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
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
-
- 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
- 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
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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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
- 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
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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
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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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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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
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US12518108P | 2008-04-22 | 2008-04-22 | |
PCT/EP2009/054724 WO2009130208A1 (en) | 2008-04-22 | 2009-04-21 | Liver-specific nucleic acid regulatory elements and methods and use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
DK2282764T3 true DK2282764T3 (da) | 2019-10-14 |
Family
ID=40790549
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK09735371.8T DK2282764T3 (da) | 2008-04-22 | 2009-04-21 | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
Country Status (8)
Country | Link |
---|---|
US (5) | US9617548B2 (da) |
EP (3) | EP3586868B1 (da) |
JP (5) | JP5797551B2 (da) |
AU (1) | AU2009240054B2 (da) |
CA (1) | CA2722238C (da) |
DK (1) | DK2282764T3 (da) |
ES (1) | ES2746907T3 (da) |
WO (1) | WO2009130208A1 (da) |
Families Citing this family (62)
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US9617548B2 (en) | 2008-04-22 | 2017-04-11 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
JP5823969B2 (ja) | 2009-10-29 | 2015-11-25 | フエー・イー・ベー・フエー・ゼツト・ウエー | 心臓特異的核酸調節因子ならびにこの方法および使用 |
US9546369B2 (en) | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のaavベースの治療 |
CA3049237C (en) | 2010-04-23 | 2024-06-11 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
US20110312001A1 (en) | 2010-06-15 | 2011-12-22 | Emile Nuwaysir | Compendium of ready-built stem cell models for interrogation of biological response |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
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 |
EP3513802B1 (en) | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
AU2014218807A1 (en) | 2013-02-22 | 2015-09-03 | Cellular Dynamics International, Inc. | Hepatocyte production via forward programming by combined genetic and chemical engineering |
WO2015012924A2 (en) * | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
EP3080143B1 (en) | 2013-12-09 | 2019-02-20 | Sangamo Therapeutics, Inc. | Methods and compositions for treating hemophilia |
SG11201605906UA (en) * | 2014-01-21 | 2016-08-30 | Univ Bruxelles | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
KR102390075B1 (ko) * | 2014-03-09 | 2022-04-26 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 오르니틴 트랜스카르바밀라아제(otc) 결핍증의 치료에 유용한 조성물 |
WO2015164228A1 (en) | 2014-04-21 | 2015-10-29 | Cellular Dynamics International, Inc. | Hepatocyte production via forward programming by combined genetic and chemical engineering |
US20170246215A1 (en) * | 2014-09-16 | 2017-08-31 | Albert Einstein College Of Medicine, Inc. | Repopulation of organs and tissues using a yap-ert2 fusion protein |
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JP6879486B2 (ja) * | 2015-03-17 | 2021-06-02 | フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel | Fviiiおよびfix用の最適化された肝臓特異的発現系 |
CA2990193A1 (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 |
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AU2016353353B2 (en) | 2015-11-13 | 2020-07-09 | Takeda Pharmaceutical Company Limited | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
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US11583563B2 (en) * | 2016-11-04 | 2023-02-21 | Takeda Pharmaceutical Company Limited | Adeno-associated virus formulations |
EP3592848A1 (en) | 2017-03-10 | 2020-01-15 | Genethon | Treatment of glycogen storage disease iii |
EP3600445A1 (en) | 2017-03-27 | 2020-02-05 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
WO2018208728A1 (en) | 2017-05-08 | 2018-11-15 | Flagship Pioneering, Inc. | Compositions for facilitating membrane fusion and uses thereof |
US10842853B2 (en) | 2017-05-22 | 2020-11-24 | Baxalta Incorporated | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B |
KR20240010098A (ko) * | 2017-05-31 | 2024-01-23 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 |
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EP4326860A1 (en) | 2021-04-20 | 2024-02-28 | Anjarium Biosciences AG | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
CN115251653B (zh) * | 2021-04-30 | 2023-10-27 | 厦门新技术集成有限公司 | 弹簧包、弹簧垫、家具和制作弹簧包的方法 |
JP2024518409A (ja) | 2021-05-07 | 2024-05-01 | アステラス インスティテュート フォー リジェネラティブ メディシン | 成熟肝細胞を作製する方法 |
AR126660A1 (es) | 2021-07-28 | 2023-11-01 | Univ Brussel Vrije | Mejorando la eficacia de la terapia génica dirigida a músculos |
CA3226119A1 (en) | 2021-08-04 | 2023-02-09 | Giuseppe RONZITTI | Hybrid promoters for gene expression in muscles and in the cns |
WO2023135273A2 (en) | 2022-01-14 | 2023-07-20 | Anjarium Biosciences Ag | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
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EP3586868B1 (en) | 2023-11-08 |
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ES2746907T3 (es) | 2020-03-09 |
US9617548B2 (en) | 2017-04-11 |
JP2020078316A (ja) | 2020-05-28 |
JP5797551B2 (ja) | 2015-10-21 |
US10471157B2 (en) | 2019-11-12 |
EP4154903A1 (en) | 2023-03-29 |
US10149914B2 (en) | 2018-12-11 |
CA2722238C (en) | 2017-11-28 |
US11654201B2 (en) | 2023-05-23 |
JP6272279B2 (ja) | 2018-01-31 |
JP2016027802A (ja) | 2016-02-25 |
EP2282764A1 (en) | 2011-02-16 |
US20110184049A1 (en) | 2011-07-28 |
US20230293728A1 (en) | 2023-09-21 |
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