AR097657A1 - Vectores de virus adenoasociados de factor viii - Google Patents
Vectores de virus adenoasociados de factor viiiInfo
- Publication number
- AR097657A1 AR097657A1 ARP140103423A ARP140103423A AR097657A1 AR 097657 A1 AR097657 A1 AR 097657A1 AR P140103423 A ARP140103423 A AR P140103423A AR P140103423 A ARP140103423 A AR P140103423A AR 097657 A1 AR097657 A1 AR 097657A1
- Authority
- AR
- Argentina
- Prior art keywords
- seq
- sequence
- construct
- established
- fviii
- 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
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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
- C07K14/435—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)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (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)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (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)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
La solicitud proporciona vectores de virus adenoasociados (AAV) de Factor VIII (FVIII), que incluyen vectores AAV de FVIII que producen un polipéptido de Factor VIII funcional y vectores AAV de FVIII con actividad de expresión alta. Reivindicación 2: El vector AAV de FVIII de la reivindicación 1, donde el ácido nucleico comprende una secuencia de nucleótidos seleccionada del grupo que consiste en: i) la secuencia Proto 1 establecida en SEQ ID Nº 1, ii) la secuencia Proto 1S establecida en SEQ ID Nº 2, iii) la secuencia Proto 25 establecida en SEQ ID Nº 3, iv) la secuencia Proto 3S establecida en SEQ ID Nº 4, v) la secuencia Proto 4 establecida en SEQ ID Nº 5, vi) la secuencia Proto 5 establecida en SEQ ID Nº 6, vii) la secuencia Proto 6 establecida en SEQ ID Nº 7, viii) la secuencia Proto 7 establecida en SEQ ID Nº 8, ix) la secuencia Construct 100ATG establecida en SEQ ID Nº 9, x) la secuencia Construct 100ATG bGH poly A establecida en SEQ ID Nº 10, xi) la secuencia Construct 100ATG short bGH poly A establecida en SEQ ID Nº 11, xii) la secuencia Construct 103ATG establecida en SEQ ID Nº 12, xiii) la secuencia Construct 103ATG short bGH poly A establecida en SEQ ID Nº 13, xiv) la secuencia Construct 105ATG bGH poly A establecida en SEQ ID Nº 14, xv) la secuencia Construct DC172ATG FVIII establecida en SEQ ID Nº 15, xvi) la secuencia Construct DC172 ATG FVIII hAAT establecida en SEQ ID Nº 16, xvii) la secuencia Construct DC172 2xHCR ATG FVIII establecida en SEQ ID Nº 17, xviii) la secuencia Construct DC172 2xHCR ATG FVIII hAAT establecida en SEQ ID Nº 18, xix) la secuencia Construct 2x SerpinAhAAT ATG FVIII establecida en SEQ ID Nº 19, xx) la secuencia Construct 2x SerpinAhAAT ATG FVIII 2x potenciador de micro-globulina establecida en SEQ ID Nº 20, xxi) la secuencia Construct 100ATG short poly A 2x potenciador de micro-globulina establecida en SEQ ID Nº 21, xxii) la secuencia Construct Factor VIII-BMN001 establecida en SEQ ID Nº 22, xxiii) la secuencia Construct Factor VIII-BMN002 establecida en SEQ ID Nº 23, xxiv) la secuencia Construct 99 establecida en SEQ ID Nº 24, xxv) la secuencia Construct 100 establecida en SEQ ID Nº 25, xxvi) la secuencia Construct 100 sentido inverso establecida en SEQ ID Nº 26, xxvii) la secuencia Construct 100AT establecida en SEQ ID Nº 27, xxviii) la secuencia Construct 100AT 2x MG establecida en SEQ ID Nº 28, xxix) la secuencia Construct 100AT 2x MG poly A establecida en SEQ ID Nº 29, xxx) la secuencia Construct 100AT 2x MG (inversa) bGH poly A establecida en SEQ ID Nº 30, xxxi) la secuencia Construct 100 bGH poly A establecida en SEQ ID Nº 31, xxxii) la secuencia Construct 100-400 establecida en SEQ ID Nº 32, xxxiii) la secuencia Construct 101 establecida en SEQ ID Nº 33, xxxiv) la secuencia Construct 102 establecida en SEQ ID Nº 34, xxxv) la secuencia Construct 103 establecida en SEQ ID Nº 35, xxxvi) la secuencia Construct 103 sentido inverso establecida en SEQ ID Nº 36, xxxvii) la secuencia Construct 103AT establecida en SEQ ID Nº 37, xxxviii) la secuencia Construct 103AT 2x MG establecida en SEQ ID Nº 38, xxxix) la secuencia Construct 103AT 2x MG poly A establecida en SEQ ID Nº 39, xl) la secuencia Construct 103 bGH poly A establecida en SEQ ID Nº 40, xli) la secuencia Construct 104 establecida en SEQ ID Nº 41, xlii) la secuencia Construct 105 establecida en SEQ ID Nº 42, xliii) la secuencia Construct 106 establecida en SEQ ID Nº 43, xliv) la secuencia Construct 106AT establecida en SEQ ID Nº 44 y xlv) la secuencia Construct 2x SerpinA hAAT establecida en SEQ ID Nº 45.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361877042P | 2013-09-12 | 2013-09-12 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR097657A1 true AR097657A1 (es) | 2016-04-06 |
Family
ID=51585244
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP140103423A AR097657A1 (es) | 2013-09-12 | 2014-09-12 | Vectores de virus adenoasociados de factor viii |
Country Status (33)
| Country | Link |
|---|---|
| US (5) | US9504762B2 (es) |
| EP (2) | EP3770169A1 (es) |
| JP (4) | JP6735672B2 (es) |
| KR (2) | KR20230006058A (es) |
| CN (2) | CN112538501A (es) |
| AR (1) | AR097657A1 (es) |
| AU (1) | AU2014318890B2 (es) |
| CA (3) | CA2921232C (es) |
| CL (1) | CL2016000502A1 (es) |
| CY (1) | 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) | MX2016003262A (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) |
| TW (1) | TWI632239B (es) |
| WO (1) | WO2015038625A1 (es) |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| HUE050484T2 (hu) | 2013-09-12 | 2020-12-28 | Biomarin Pharm Inc | VIII. faktort kódoló gént tartalmazó AAV vektorok |
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| AU2016232146B2 (en) * | 2015-03-17 | 2021-11-04 | 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 |
| US10058624B2 (en) * | 2015-04-16 | 2018-08-28 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| TWI756185B (zh) * | 2015-09-24 | 2022-03-01 | 美商拜奧馬林製藥公司 | 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 |
| PE20181206A1 (es) * | 2015-10-28 | 2018-07-23 | Sangamo Therapeutics Inc | Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos |
| 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 |
| JP6695426B2 (ja) * | 2015-11-13 | 2020-05-20 | バクスアルタ インコーポレイテッド | 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター |
| 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 |
| WO2017180857A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
| US11427809B2 (en) | 2016-05-25 | 2022-08-30 | Lonza Houston, Inc. | Methods for isolating adeno-associated virus using a polydialkylammonium salt |
| EP3491008A2 (en) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical Inc. | Novel adeno-associated virus capsid proteins |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3071519A1 (en) * | 2017-08-01 | 2019-02-07 | Spark Therapeutics, Inc. | Factor viii (fviii) gene therapy methods |
| EP3790627A2 (en) | 2018-05-09 | 2021-03-17 | BioMarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| MX2020012167A (es) | 2018-05-14 | 2022-07-13 | Biomarin Pharm Inc | Expresion estable de vectores de aav en sujetos juveniles. |
| 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 |
| US20220333135A1 (en) | 2018-07-16 | 2022-10-20 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| EP3799569A1 (en) * | 2018-07-27 | 2021-04-07 | Zentrum für Forschungsförderung in der Pädiatrie GmbH | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| CN113194982A (zh) * | 2018-10-12 | 2021-07-30 | 建新公司 | 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生 |
| TW202039546A (zh) * | 2019-01-16 | 2020-11-01 | 美商巴克斯歐塔公司 | 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 |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| SG11202109850SA (en) * | 2019-03-13 | 2021-10-28 | 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 |
| WO2021084275A1 (en) * | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Factor viii polypeptide |
| 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 |
| CA3196778A1 (en) | 2020-11-02 | 2022-05-05 | Biomarin Pharmaceutical, Inc. | Process for enriching adeno-associated virus |
| EP4355768A1 (en) | 2021-06-14 | 2024-04-24 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| WO2022269466A1 (en) * | 2021-06-22 | 2022-12-29 | Pfizer Inc. | Production of adeno-associated virus vector in insect cells |
| AU2022317647A1 (en) * | 2021-07-28 | 2024-01-25 | Biomarin Pharmaceutical Inc. | Large scale adeno-associated virus production systems |
| KR20240049332A (ko) * | 2021-08-23 | 2024-04-16 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 인자 viii 유전자 |
| WO2023036054A2 (en) * | 2021-09-08 | 2023-03-16 | Inspirar Limited | Composition and method for treating hemophilia |
| 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 |
| WO2023173078A2 (en) * | 2022-03-11 | 2023-09-14 | Homology Medicines, Inc. | Bidirectional dual promoter expression vectors and uses thereof |
| WO2023205300A2 (en) * | 2022-04-20 | 2023-10-26 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
| WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic 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 |
| WO2024074143A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| CN116555266A (zh) * | 2023-06-09 | 2023-08-08 | 呈诺再生医学科技(北京)有限公司 | 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用 |
| CN118064436A (zh) * | 2024-04-18 | 2024-05-24 | 上海凌医生物科技有限公司 | 一种增强基因表达的嵌合启动子 |
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| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| SK282843B6 (sk) | 1993-07-13 | 2002-12-03 | Rhone-Poulenc Rorer S. A. | Defektný rekombinantný adenovírus a farmaceutický prostriedok s jeho obsahom |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| JP2001500376A (ja) | 1996-09-06 | 2001-01-16 | カイロン コーポレイション | 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物 |
| US6383794B1 (en) * | 1998-08-24 | 2002-05-07 | Uab Research Foundation | Methods of producing high titer recombinant adeno-associated virus |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| 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 |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| EP2438931B1 (en) | 2004-09-22 | 2013-11-13 | St. Jude Children's Research Hospital | Improved expression of factor IX in gene therapy vectors |
| CN1286981C (zh) * | 2004-11-30 | 2006-11-29 | 华中科技大学同济医学院附属同济医院 | 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法 |
| 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 |
| 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 |
| HUE050484T2 (hu) | 2013-09-12 | 2020-12-28 | Biomarin Pharm Inc | VIII. faktort kódoló gént tartalmazó AAV vektorok |
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