RU2018136611A3 - - Google Patents
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- RU2018136611A3 RU2018136611A3 RU2018136611A RU2018136611A RU2018136611A3 RU 2018136611 A3 RU2018136611 A3 RU 2018136611A3 RU 2018136611 A RU2018136611 A RU 2018136611A RU 2018136611 A RU2018136611 A RU 2018136611A RU 2018136611 A3 RU2018136611 A3 RU 2018136611A3
- Authority
- RU
- Russia
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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
- 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
- A61K48/0075—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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
-
- 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
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Dispersion Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323336P | 2016-04-15 | 2016-04-15 | |
| US62/323,336 | 2016-04-15 | ||
| US201662331807P | 2016-05-04 | 2016-05-04 | |
| US62/331,807 | 2016-05-04 | ||
| US201662428866P | 2016-12-01 | 2016-12-01 | |
| US62/428,866 | 2016-12-01 | ||
| PCT/US2017/027396 WO2017180857A1 (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2018136611A RU2018136611A (ru) | 2020-05-15 |
| RU2018136611A3 true RU2018136611A3 (ru) | 2020-08-11 |
| RU2762257C2 RU2762257C2 (ru) | 2021-12-17 |
Family
ID=59253992
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018136611A RU2762257C2 (ru) | 2016-04-15 | 2017-04-13 | Генная терапия для лечения гемофилии a |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US11779656B2 (ru) |
| EP (1) | EP3452102B9 (ru) |
| JP (2) | JP7347933B2 (ru) |
| KR (1) | KR102450833B1 (ru) |
| CN (1) | CN109562191A (ru) |
| AU (1) | AU2017248659B2 (ru) |
| BR (1) | BR112018071200A2 (ru) |
| CA (1) | CA3019425A1 (ru) |
| IL (1) | IL262215B2 (ru) |
| MX (1) | MX2018012537A (ru) |
| PE (2) | PE20181922A1 (ru) |
| RU (1) | RU2762257C2 (ru) |
| SA (1) | SA518400233B1 (ru) |
| SG (2) | SG10202009849WA (ru) |
| TW (1) | TWI791433B (ru) |
| WO (1) | WO2017180857A1 (ru) |
| ZA (1) | ZA201806477B (ru) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3252157A1 (en) | 2008-09-15 | 2017-12-06 | Paolo Simioni | Factor ix polypeptide mutant, its uses and a method for its production |
| WO2017087395A1 (en) | 2015-11-16 | 2017-05-26 | Research Institute At Nationwide Children's Hospital | Materials and methods for treatment of titin-based myopathies and other titinopaties |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| RU2762257C2 (ru) * | 2016-04-15 | 2021-12-17 | Зе Трастис Оф Зе Юниверсити Оф Пенсильвания | Генная терапия для лечения гемофилии a |
| CA3089080A1 (en) | 2018-01-31 | 2019-08-08 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| CN112543810A (zh) | 2018-06-29 | 2021-03-23 | 全国儿童医院研究所 | 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法 |
| JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
| CN120349985A (zh) * | 2018-10-12 | 2025-07-22 | 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 |
| KR20210075994A (ko) * | 2018-10-15 | 2021-06-23 | 리젠엑스바이오 인크. | 복제 결함 바이러스 벡터 및 바이러스의 감염성을 측정하는 방법 |
| PL3867372T3 (pl) | 2018-10-16 | 2025-06-23 | Blueallele Corporation | Sposoby ukierunkowanego wprowadzania dna do genów |
| GB201818816D0 (en) * | 2018-11-19 | 2019-01-02 | Synpromics Ltd | Regulatory nucleic acid sequences |
| 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 |
| US12377170B2 (en) * | 2019-02-26 | 2025-08-05 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy |
| MA55313A (fr) * | 2019-03-13 | 2022-01-19 | Generation Bio Co | Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii |
| BR112022003142A2 (pt) | 2019-08-21 | 2022-05-17 | Res Inst Nationwide Childrens Hospital | Entrega de vetor de vírus adenoassociado de alfa-sarcoglicano e tratamento de distrofia muscular |
| JP2023501263A (ja) * | 2019-11-01 | 2023-01-18 | フリーライン セラピューティクス リミテッド | 第ｖｉｉｉ因子ポリペプチド |
| CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技（武汉）有限公司 | 一种肝脏特异性启动子及其应用 |
| PE20231100A1 (es) * | 2020-01-16 | 2023-07-18 | Takeda Pharmaceuticals Co | Terapia genica basada en virus adenoasociados para la fenilcetonuria |
| WO2021231579A1 (en) | 2020-05-12 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2022165245A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
| EP4284442A4 (en) * | 2021-02-01 | 2025-04-23 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2023078220A1 (zh) * | 2021-11-02 | 2023-05-11 | 苏州华毅乐健生物科技有限公司 | 分离的核酸分子及其应用 |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250276093A1 (en) * | 2022-04-20 | 2025-09-04 | AskBio Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| WO2023240222A2 (en) * | 2022-06-10 | 2023-12-14 | University Of Florida Research Foundation, Incorporated | Immune tolerance induction to viral capsids |
Family Cites Families (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US5661008A (en) | 1991-03-15 | 1997-08-26 | Kabi Pharmacia Ab | Recombinant human factor VIII derivatives |
| US5439824A (en) | 1993-05-06 | 1995-08-08 | The United States Of America | Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II |
| US6268212B1 (en) | 1993-10-18 | 2001-07-31 | Amgen Inc. | Tissue specific transgene expression |
| US6818439B1 (en) | 1994-12-30 | 2004-11-16 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders |
| EP0951544A2 (en) * | 1996-07-03 | 1999-10-27 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of human disease |
| EP1036177A1 (en) | 1997-12-05 | 2000-09-20 | The Immune Response Corporation | Novel vectors and genes exhibiting increased expression |
| US6783961B1 (en) | 1999-02-26 | 2004-08-31 | Genset S.A. | Expressed sequence tags and encoded human proteins |
| US6797505B2 (en) | 1998-05-27 | 2004-09-28 | Cell Genesys, Inc. | Recombinant AAV vectors for gene therapy of hemophilia A |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| EP1038959A1 (en) | 1999-03-17 | 2000-09-27 | Aventis Behring Gesellschaft mit beschränkter Haftung | Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX |
| US6610906B1 (en) | 1999-06-09 | 2003-08-26 | The Regents Of The University Of Michigan | Nucleotide sequences for gene regulation and methods of use thereof |
| EP1224312A1 (en) | 1999-10-12 | 2002-07-24 | The University of North Carolina at Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| EP1818407A3 (en) | 1999-11-16 | 2007-08-29 | Genzyme Corporation | Improved regulatory elements for delivery to the liver |
| MXPA02009221A (es) | 2000-03-22 | 2005-07-25 | Octagene Gmbh | Produccion de factores de coagulacion sanguineo recombinantes en lineas celular humanas. |
| US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US7351813B2 (en) | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
| US6812339B1 (en) | 2000-09-08 | 2004-11-02 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
| CA2441454A1 (en) | 2001-03-14 | 2002-09-19 | Avigen, Inc. | Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions |
| US8021875B2 (en) | 2001-08-27 | 2011-09-20 | Roche Madison Inc. | Methods for expression of transgenes |
| AU2002337901B2 (en) | 2001-10-05 | 2008-01-17 | Expression Therapeutics Llc | Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use |
| PL217623B1 (pl) | 2001-11-13 | 2014-08-29 | Univ Pennsylvania | Sposób wykrywania sekwencji wirusa stowarzyszonego z adenowirusem (AAV) w próbce |
| FI3517134T3 (fi) | 2001-12-17 | 2024-04-03 | Univ Pennsylvania | Adeno-assosioituneen viruksen (aav) serotyypin 8 sekvenssejä, niitä sisältäviä vektoreita ja niiden käyttöjä |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| DK2292780T3 (en) | 2003-09-30 | 2017-12-04 | Univ Pennsylvania | Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof |
| BRPI0415296A (pt) | 2003-10-16 | 2006-12-26 | Claude Negrier | cdna modificado para os nìveis de expressão elevados do fator viii e seus derivados |
| EP1699469B1 (en) | 2003-12-03 | 2010-04-07 | University of Rochester | Recombinant factor viii having increased specific activity |
| ES2381796T3 (es) | 2004-09-22 | 2012-05-31 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| US8008468B2 (en) | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| WO2006102072A2 (en) | 2005-03-23 | 2006-09-28 | The Trustees Of The University Of Pennsylvania | Use of a pa131 polypeptide in treatment of atherosclerosis |
| AU2006233638A1 (en) | 2005-04-14 | 2006-10-19 | Csl Behring Gmbh | Modified coagulation Factor VIII with enhanced stability and its derivates |
| WO2007016331A2 (en) | 2005-07-29 | 2007-02-08 | Children's Hospital Medical Center | Classification and diagnosis of the molecular basis of cholestasis |
| 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 |
| JP2009519710A (ja) | 2005-12-16 | 2009-05-21 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 遺伝子発現調節エレメントのハイスループットでの特徴付けのための機能性アレイ |
| US9150882B2 (en) | 2006-01-31 | 2015-10-06 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| WO2007120533A2 (en) * | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| US8198421B2 (en) | 2006-06-19 | 2012-06-12 | Asklepios Biopharmaceutical, Inc. | Modified factor VIII and factor IX genes and vectors for gene therapy |
| EP2097538A4 (en) | 2006-12-07 | 2011-11-30 | Switchgear Genomics | TRANSCRIPTION REAGULATION ELEMENTS OF BIOLOGICAL PATHS, TOOLS AND METHODS |
| EP3231440A1 (en) | 2006-12-22 | 2017-10-18 | CSL Behring GmbH | Modified coagulation factors with prolonged in vivo half-life |
| US20100120628A1 (en) | 2007-02-06 | 2010-05-13 | Genizon Biosciences Inc. | Genemap of the human genes associated with adhd |
| US8309698B2 (en) | 2008-02-14 | 2012-11-13 | Mogam Biotechnology Research Institute | Expression vector suitable for expression of a coding sequence for gene therapy |
| KR100981092B1 (ko) | 2008-02-29 | 2010-09-08 | 고려대학교 산학협력단 | 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합발현 벡터 시스템 |
| WO2009122401A2 (en) | 2008-04-03 | 2009-10-08 | Yeda Research And Development Co. Ltd. | Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same |
| JP5797551B2 (ja) | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| WO2009158513A1 (en) | 2008-06-25 | 2009-12-30 | Source Precision Medicine, Inc. | Gene expression profiling for identification, monitoring, and treatment of osteoarthritis |
| KR101034811B1 (ko) | 2008-08-25 | 2011-05-16 | 단국대학교 산학협력단 | 조직 특이적 프로모터와 암 특이 유전자를 타겟팅하는트랜스-스플라이싱 라이보자임을 포함하는 재조합아데노바이러스 및 이의 용도 |
| CA2756596A1 (en) | 2009-03-27 | 2010-09-30 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| CN101935674A (zh) * | 2009-06-30 | 2011-01-05 | 中国医学科学院血液学研究所 | 一种用于血友病b基因治疗的腺病毒载体及其用途 |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| CA2791974A1 (en) | 2010-03-01 | 2011-09-09 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the hcv genome |
| AU2011238708B2 (en) | 2010-03-29 | 2016-02-11 | The Trustees Of The University Of Pennsylvania | Pharmacologically Induced Transgene Ablation system |
| US9315825B2 (en) * | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| NZ607690A (en) * | 2010-09-02 | 2014-10-31 | Molmed Spa | Stable production of lentiviral vectors |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia 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 |
| 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 |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| US10308705B2 (en) * | 2015-02-06 | 2019-06-04 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor VIII gene expression cassettes and their use |
| US20180110877A1 (en) * | 2015-04-27 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE |
| TWI695069B (zh) * | 2015-11-13 | 2020-06-01 | 美商百克莎塔股份有限公司 | 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 |
| EP3992283A1 (en) | 2015-12-11 | 2022-05-04 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| RU2762257C2 (ru) * | 2016-04-15 | 2021-12-17 | Зе Трастис Оф Зе Юниверсити Оф Пенсильвания | Генная терапия для лечения гемофилии a |
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2017
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2018
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2019
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2022
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2023
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