PE20160769A1 - Vectores del factor viii del virus adeno-asociado - Google Patents
Vectores del factor viii del virus adeno-asociadoInfo
- Publication number
- PE20160769A1 PE20160769A1 PE2016000353A PE2016000353A PE20160769A1 PE 20160769 A1 PE20160769 A1 PE 20160769A1 PE 2016000353 A PE2016000353 A PE 2016000353A PE 2016000353 A PE2016000353 A PE 2016000353A PE 20160769 A1 PE20160769 A1 PE 20160769A1
- Authority
- PE
- Peru
- Prior art keywords
- vectors
- factor viii
- associated virus
- adeno
- aav
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
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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
-
- 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
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Hematology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (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)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente invencion se refiere a vectores mejorados del Factor VIII (FVIII) del virus asociado a adenovirus (AAV), que incluyen vectores del FVIII del AAV que producen un polipeptido del Factor VIII funcional y vectores del FVIII del AAV con alta actividad de expresion, y composiciones que los contienen utiles para tratar la hemofilia A
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877042P | 2013-09-12 | 2013-09-12 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20160769A1 true PE20160769A1 (es) | 2016-08-12 |
Family
ID=51585244
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2016000353A PE20160769A1 (es) | 2013-09-12 | 2014-09-10 | Vectores del factor viii del virus adeno-asociado |
Country Status (34)
| Country | Link |
|---|---|
| US (5) | US9504762B2 (es) |
| EP (2) | EP3044231B1 (es) |
| JP (4) | JP6735672B2 (es) |
| KR (2) | KR20230006058A (es) |
| CN (2) | CN112538501A (es) |
| AR (1) | AR097657A1 (es) |
| AU (1) | AU2014318890B2 (es) |
| CA (3) | CA3178379A1 (es) |
| CL (1) | CL2016000502A1 (es) |
| CY (2) | CY1124213T1 (es) |
| DK (1) | DK3044231T3 (es) |
| ES (1) | ES2813698T3 (es) |
| FI (1) | FIC20230007I1 (es) |
| FR (1) | FR23C1007I2 (es) |
| HK (1) | HK1226416A1 (es) |
| HR (1) | HRP20201324T1 (es) |
| HU (2) | HUE050484T2 (es) |
| IL (2) | IL244404B (es) |
| LT (2) | LT3044231T (es) |
| MX (2) | MX386802B (es) |
| NL (1) | NL301222I2 (es) |
| NO (1) | NO2023003I1 (es) |
| NZ (1) | NZ716549A (es) |
| PE (1) | PE20160769A1 (es) |
| PH (1) | PH12016500328A1 (es) |
| PL (1) | PL3044231T3 (es) |
| PT (1) | PT3044231T (es) |
| RS (1) | RS61039B1 (es) |
| RU (1) | RU2710307C2 (es) |
| SG (1) | SG11201601932UA (es) |
| SI (1) | SI3044231T1 (es) |
| SM (1) | SMT202000458T1 (es) |
| TW (1) | TWI632239B (es) |
| WO (1) | WO2015038625A1 (es) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| LT3044231T (lt) | 2013-09-12 | 2020-11-10 | Biomarin Pharmaceutical Inc. | Aav vektoriai, apimantys viii faktorių koduojantį geną |
| BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| US11007280B2 (en) | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP3283126B1 (en) | 2015-04-16 | 2019-11-06 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| BR112018006074A2 (pt) * | 2015-09-24 | 2018-10-09 | Biomarin Pharm Inc | vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas |
| PH12018500927B1 (en) * | 2015-10-28 | 2024-07-03 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| MY190067A (en) * | 2015-11-13 | 2022-03-24 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| TWI850587B (zh) * | 2015-11-13 | 2024-08-01 | 日商武田藥品工業股份有限公司 | 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 |
| SG11201808422QA (en) * | 2016-04-15 | 2018-10-30 | Univ Pennsylvania | Gene therapy for treating hemophilia a |
| AU2017269371B2 (en) | 2016-05-25 | 2021-08-05 | Lonza Houston Inc. | Methods for isolating adeno-associated virus using a polydiallyldialkylammonium salt |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR20200066289A (ko) * | 2017-08-01 | 2020-06-09 | 스파크 테라퓨틱스, 인코포레이티드 | 인자 viii (fviii) 유전자 요법 방법 |
| CA3099704A1 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202016298A (zh) | 2018-05-14 | 2020-05-01 | 美商拜奧馬林製藥公司 | Aav 載體於幼年受試者中之穩定表現 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019306194A1 (en) | 2018-07-16 | 2021-02-04 | Baxalta GmbH | Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression |
| AU2018434176A1 (en) * | 2018-07-27 | 2021-01-28 | Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| SG11202103614RA (en) * | 2018-10-12 | 2021-05-28 | Genzyme Corp | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| CA3133255A1 (en) * | 2019-03-13 | 2020-09-17 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| JP6831943B1 (ja) | 2019-10-24 | 2021-02-17 | 三ツ星ベルト株式会社 | 摩擦伝動ベルトおよびその製造方法 |
| CN114901686A (zh) * | 2019-11-01 | 2022-08-12 | 自由行疗法有限公司 | 因子viii多肽 |
| BR112022009279A2 (pt) * | 2019-11-14 | 2022-09-06 | Biomarin Pharm Inc | Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado |
| WO2021119357A2 (en) | 2019-12-12 | 2021-06-17 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
| WO2021262963A1 (en) | 2020-06-24 | 2021-12-30 | Bioverativ Therapeutics Inc. | Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein |
| AU2021372262A1 (en) | 2020-11-02 | 2023-06-01 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| WO2022264040A1 (en) | 2021-06-14 | 2022-12-22 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| EP4359547A1 (en) * | 2021-06-22 | 2024-05-01 | Pfizer Inc. | Production of adeno-associated virus vector in insect cells |
| IL310039A (en) * | 2021-07-28 | 2024-03-01 | Biomarin Pharm Inc | Large scale adeno-associated virus production systems |
| AR126846A1 (es) * | 2021-08-23 | 2023-11-22 | Bioverativ Therapeutics Inc | Genes del factor viii optimizados |
| WO2023036054A2 (en) * | 2021-09-08 | 2023-03-16 | Inspirar Limited | Composition and method for treating hemophilia |
| JP2025504404A (ja) | 2022-01-14 | 2025-02-12 | アンジャリウム バイオサイエンシズ エージー | Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法 |
| AR128760A1 (es) * | 2022-03-11 | 2024-06-12 | Homology Medicines Inc | Vectores de expresión con promotores dobles bidireccionales y sus usos |
| EP4511055A2 (en) * | 2022-04-20 | 2025-02-26 | AskBio Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| TW202421788A (zh) | 2022-09-22 | 2024-06-01 | 美商拜奧馬林製藥公司 | 用ａａｖ基因療法載體治療致心律不整性心肌病 |
| WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
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| EP1739179A1 (en) * | 2005-06-30 | 2007-01-03 | Octapharma AG | Serum-free stable transfection and production of recombinant human proteins in human cell lines |
| 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 |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| EP2768521B1 (en) * | 2011-10-18 | 2016-07-13 | CSL Behring GmbH | Combined use of a sulfated glycosaminoglycan and a hyaluronidase for improving the bioavailability of factor viii |
| CN103215308B (zh) * | 2013-02-01 | 2015-12-23 | 中国科学院苏州生物医学工程技术研究所 | 表达重组人fviii的整合质粒、细胞株及其构建方法和应用 |
| LT3044231T (lt) * | 2013-09-12 | 2020-11-10 | Biomarin Pharmaceutical Inc. | Aav vektoriai, apimantys viii faktorių koduojantį geną |
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