RU2018114888A3 - - Google Patents
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- RU2018114888A3 RU2018114888A3 RU2018114888A RU2018114888A RU2018114888A3 RU 2018114888 A3 RU2018114888 A3 RU 2018114888A3 RU 2018114888 A RU2018114888 A RU 2018114888A RU 2018114888 A RU2018114888 A RU 2018114888A RU 2018114888 A3 RU2018114888 A3 RU 2018114888A3
- Authority
- RU
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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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6854—Immunoglobulins
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16041—Use of virus, viral particle or viral elements as a vector
- C12N2710/16044—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
-
- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
- G01N2333/01—DNA viruses
- G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Hematology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Wood Science & Technology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Urology & Nephrology (AREA)
- Biophysics (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- General Engineering & Computer Science (AREA)
- Inorganic Chemistry (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562232242P | 2015-09-24 | 2015-09-24 | |
| US62/232,242 | 2015-09-24 | ||
| US201662323182P | 2016-04-15 | 2016-04-15 | |
| US62/323,182 | 2016-04-15 | ||
| US201662365544P | 2016-07-22 | 2016-07-22 | |
| US62/365,544 | 2016-07-22 | ||
| PCT/US2016/053269 WO2017053677A1 (en) | 2015-09-24 | 2016-09-23 | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020126034A Division RU2020126034A (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2018114888A3 true RU2018114888A3 (enExample) | 2019-10-24 |
| RU2018114888A RU2018114888A (ru) | 2019-10-24 |
| RU2742352C2 RU2742352C2 (ru) | 2021-02-05 |
Family
ID=57113749
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020126034A RU2020126034A (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| RU2018114888A RU2742352C2 (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020126034A RU2020126034A (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US10512675B2 (enExample) |
| EP (1) | EP3352787A1 (enExample) |
| JP (3) | JP2018535929A (enExample) |
| KR (1) | KR102805910B1 (enExample) |
| CN (1) | CN108778323A (enExample) |
| AU (2) | AU2016326602A1 (enExample) |
| BR (1) | BR112018006074A2 (enExample) |
| CA (1) | CA2999297A1 (enExample) |
| CL (1) | CL2018000740A1 (enExample) |
| IL (2) | IL297353B2 (enExample) |
| MA (1) | MA42934A (enExample) |
| MX (1) | MX2018003702A (enExample) |
| PE (2) | PE20241346A1 (enExample) |
| PH (1) | PH12018500594A1 (enExample) |
| RU (2) | RU2020126034A (enExample) |
| TW (3) | TW202446416A (enExample) |
| WO (1) | WO2017053677A1 (enExample) |
| ZA (1) | ZA201801657B (enExample) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| JP2018535929A (ja) * | 2015-09-24 | 2018-12-06 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 |
| US10143760B2 (en) | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| JP6994018B2 (ja) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| US11452749B2 (en) | 2017-07-10 | 2022-09-27 | Uniqure Ip B.V. | Means and methods for AAV gene therapy in humans |
| US20200237930A1 (en) * | 2017-08-01 | 2020-07-30 | Spark Therapeutics, Inc. | Factor viii (fviii) gene therapy methods |
| CN111936621B (zh) * | 2018-02-05 | 2025-05-13 | 奥登茨治疗公司 | 转录调控元件和它的用途 |
| US20210093735A1 (en) * | 2018-04-26 | 2021-04-01 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
| 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 |
| CN112424345A (zh) * | 2018-05-14 | 2021-02-26 | 生物马林药物股份有限公司 | Aav载体在青少年对象中的稳定表达 |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| US12091675B2 (en) * | 2018-07-16 | 2024-09-17 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression |
| AU2019314513B2 (en) * | 2018-08-03 | 2026-04-30 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| CN113226352B (zh) * | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| KR20210151785A (ko) | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| JP7853100B2 (ja) | 2019-06-05 | 2026-04-28 | オーチャード セラピューティクス（ヨーロッパ）リミテッド | 真核細胞を修飾するための組成物及び方法 |
| CN114375205A (zh) * | 2019-06-20 | 2022-04-19 | 武田药品工业株式会社 | 用基于病毒的基因疗法进行治疗的方法 |
| WO2021084276A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Factor viii construct |
| AU2020395323A1 (en) * | 2019-12-06 | 2022-06-30 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating factor VIII function |
| CN114981299A (zh) * | 2019-12-12 | 2022-08-30 | 武田药品工业株式会社 | 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法 |
| KR102167829B1 (ko) * | 2020-02-10 | 2020-10-20 | 주식회사 이노테라피 | 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US12540333B2 (en) * | 2020-11-02 | 2026-02-03 | Regeneron Pharmaceuticals, Inc. | Characterization of impurities in adeno-associated virus (AAV) samples and formulation compositions to stabilize AAV |
| KR20230078805A (ko) | 2020-11-02 | 2023-06-02 | 바이오마린 파머수티컬 인크. | 아데노-연관 바이러스의 농축 공정 |
| CN116350801A (zh) | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2025045082A1 (zh) * | 2023-09-01 | 2025-03-06 | 康霖生物科技（杭州）有限公司 | 一种缓冲系统及其相应液体制剂及其用途 |
| WO2026019230A1 (ko) * | 2024-07-15 | 2026-01-22 | 삼성바이오에피스 주식회사 | 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템 |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| EP0933997A4 (en) | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS |
| WO1999061595A2 (en) | 1998-05-27 | 1999-12-02 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated expression of factor viii activity |
| 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 |
| ES2436741T3 (es) | 2004-09-22 | 2014-01-07 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| 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 |
| CA2703948A1 (en) * | 2007-11-01 | 2009-05-07 | University Of Rochester | Recombinant factor viii having increased stability |
| 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 |
| KR101337797B1 (ko) * | 2010-07-14 | 2013-12-06 | 한미사이언스 주식회사 | 지속형 인간 성장 호르몬 결합체 액상 제제 |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| US20140003880A1 (en) * | 2012-06-30 | 2014-01-02 | General Electric Company | Ceramic matrix composite and metal attachment configurations |
| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| ES2813698T3 (es) | 2013-09-12 | 2021-03-24 | Biomarin Pharm Inc | Vectores de AAV que comprenden un gen que codifica el factor VIII |
| EA201791803A1 (ru) | 2015-02-10 | 2017-11-30 | Джензим Корпорейшн | Улучшенная доставка вирусных частиц в полосатое тело и кору больших полушарий |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| JP2018535929A (ja) * | 2015-09-24 | 2018-12-06 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 |
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