BR112018071200A2 - terapia genética para o tratamento da hemofilia a - Google Patents
terapia genética para o tratamento da hemofilia aInfo
- Publication number
- BR112018071200A2 BR112018071200A2 BR112018071200-0A BR112018071200A BR112018071200A2 BR 112018071200 A2 BR112018071200 A2 BR 112018071200A2 BR 112018071200 A BR112018071200 A BR 112018071200A BR 112018071200 A2 BR112018071200 A2 BR 112018071200A2
- Authority
- BR
- Brazil
- Prior art keywords
- hemophilia
- treatment
- gene therapy
- compositions
- raav
- Prior art date
Links
- 208000031220 Hemophilia Diseases 0.000 title abstract 2
- 208000009292 Hemophilia A Diseases 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 1
- 108010071690 Prealbumin Proteins 0.000 abstract 1
- 102000009190 Transthyretin Human genes 0.000 abstract 1
- 239000003623 enhancer Substances 0.000 abstract 1
- 102000057593 human F8 Human genes 0.000 abstract 1
- 229960000900 human factor viii Drugs 0.000 abstract 1
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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Dermatology (AREA)
- Immunology (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
composições e regimes úteis no tratamento da hemofilia a são proporcionados. as composições incluem vírus adeno-associados recombinantes (raav) com um potenciador de transtirretina e promotor da expressão dirigida de um fator viii humano.
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 (1)
Publication Number | Publication Date |
---|---|
BR112018071200A2 true BR112018071200A2 (pt) | 2019-04-09 |
Family
ID=59253992
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112018071200-0A BR112018071200A2 (pt) | 2016-04-15 | 2017-04-13 | terapia genética para o tratamento da hemofilia a |
Country Status (16)
Country | Link |
---|---|
US (3) | US11779656B2 (pt) |
EP (1) | EP3452102B1 (pt) |
JP (2) | JP7347933B2 (pt) |
KR (1) | KR102450833B1 (pt) |
CN (1) | CN109562191A (pt) |
AU (1) | AU2017248659B2 (pt) |
BR (1) | BR112018071200A2 (pt) |
CA (1) | CA3019425A1 (pt) |
IL (1) | IL262215B2 (pt) |
MX (1) | MX2018012537A (pt) |
PE (1) | PE20181922A1 (pt) |
RU (1) | RU2762257C2 (pt) |
SA (1) | SA518400233B1 (pt) |
SG (2) | SG11201808422QA (pt) |
TW (1) | TWI791433B (pt) |
WO (1) | WO2017180857A1 (pt) |
Families Citing this family (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
CA3115524A1 (en) * | 2018-10-15 | 2020-04-23 | Win Den Cheung | Methods for measuring the infectivity of replication defective viral vectors and viruses |
EP3930765A1 (en) * | 2019-02-26 | 2022-01-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
KR20210151785A (ko) * | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
EP4051703A1 (en) * | 2019-11-01 | 2022-09-07 | Freeline Therapeutics Limited | Factor viii polypeptide |
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 |
JP2023526310A (ja) | 2020-05-12 | 2023-06-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 導入遺伝子発現のdrg特異的低減のための組成物 |
WO2022165246A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
WO2022165421A1 (en) * | 2021-02-01 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
CA3209779A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2023205300A2 (en) * | 2022-04-20 | 2023-10-26 | Asklepios Biopharmaceutical, 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 (64)
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 |
CA2312291A1 (en) | 1997-12-05 | 1999-06-17 | 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 |
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 |
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 |
JP2003511082A (ja) | 1999-10-12 | 2003-03-25 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 第viii因子をコードするアデノ随伴ウィルスベクターとその使用法 |
ATE360082T1 (de) | 1999-11-16 | 2007-05-15 | Genzyme Corp | Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber |
DE60115613T2 (de) | 2000-03-22 | 2006-08-24 | Octagene Gmbh | Herstellung von rekombinanten muteine des blutgerinnungsfaktors viii in humanen zellinien |
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 |
JP2004532822A (ja) | 2001-03-14 | 2004-10-28 | アビジェン, インコーポレイテッド | ビリオンの管逆行感染による組換えアデノ随伴ウイルス媒介遺伝子移入 |
US8021875B2 (en) | 2001-08-27 | 2011-09-20 | Roche Madison Inc. | Methods for expression of transgenes |
CA2461443C (en) | 2001-10-05 | 2011-07-12 | Emory University | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
US20080312143A1 (en) | 2003-10-16 | 2008-12-18 | Zlb Behring Gmbh | Modified Cdna for High Expression Levels of Factor VIII and Its Derivatives |
WO2005055930A2 (en) | 2003-12-03 | 2005-06-23 | University Of Rochester | Recombinant factor viii having increased specific activity |
DK1804839T3 (da) | 2004-09-22 | 2012-04-10 | St Jude Childrens Res Hospital | Forbedret ekspression af faktor ix i genterapivektorer |
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 |
CA2604299A1 (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 |
WO2007078599A2 (en) | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
EP1979485A2 (en) | 2006-01-31 | 2008-10-15 | 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 |
WO2007149852A2 (en) | 2006-06-19 | 2007-12-27 | 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 |
WO2008077616A1 (en) | 2006-12-22 | 2008-07-03 | Csl Behring Gmbh | Modified coagulation factors with prolonged in vivo half-life |
EP2118320A4 (en) | 2007-02-06 | 2010-05-19 | Genizon Biosciences Inc | GENE CARD OF HUMAN GENES ASSOCIATED WITH HYPERACTIVITY ATTENTION DEFICIT DISORDER (ADHD) |
KR101190591B1 (ko) | 2008-02-14 | 2012-10-15 | 재단법인 목암생명공학연구소 | 유전자 치료용 코딩 서열의 발현에 적합한 발현 벡터 |
KR100981092B1 (ko) | 2008-02-29 | 2010-09-08 | 고려대학교 산학협력단 | 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합발현 벡터 시스템 |
EP2271763A2 (en) | 2008-04-03 | 2011-01-12 | Yeda Research And Development Company Ltd. | Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same |
DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
AU2009262104A1 (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 |
WO2010109053A1 (es) | 2009-03-27 | 2010-09-30 | Proyeto De Biomedicina Cima, S.L. | Métodos y composiciones para el tratamiento de cirrosis y fibrosis hepática |
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 |
WO2011109380A1 (en) | 2010-03-01 | 2011-09-09 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the hcv genome |
CA2793633A1 (en) | 2010-03-29 | 2011-10-13 | 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 |
PL2480678T3 (pl) * | 2010-09-02 | 2014-08-29 | Molmed Spa | Stabilna produkcja wektorów lentiwirusowych |
FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
US10398787B2 (en) * | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
EP3770169A1 (en) * | 2013-09-12 | 2021-01-27 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
EP3611186A1 (en) * | 2015-02-06 | 2020-02-19 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
JP6851319B2 (ja) * | 2015-04-27 | 2021-03-31 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系 |
TWI777175B (zh) * | 2015-11-13 | 2022-09-11 | 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 |
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2017
- 2017-04-13 CA CA3019425A patent/CA3019425A1/en active Pending
- 2017-04-13 US US16/093,798 patent/US11779656B2/en active Active
- 2017-04-13 MX MX2018012537A patent/MX2018012537A/es unknown
- 2017-04-13 TW TW106112302A patent/TWI791433B/zh active
- 2017-04-13 PE PE2018001994A patent/PE20181922A1/es unknown
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
- 2017-04-13 RU RU2018136611A patent/RU2762257C2/ru active
- 2017-04-13 AU AU2017248659A patent/AU2017248659B2/en active Active
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/ja active Active
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en active Application Filing
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/pt unknown
- 2017-04-13 EP EP17734159.1A patent/EP3452102B1/en active Active
- 2017-04-13 CN CN201780023787.5A patent/CN109562191A/zh active Pending
- 2017-04-13 SG SG10202009849WA patent/SG10202009849WA/en unknown
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/ko active IP Right Grant
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2018
- 2018-10-08 IL IL262215A patent/IL262215B2/en unknown
- 2018-10-14 SA SA518400233A patent/SA518400233B1/ar unknown
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2019
- 2019-11-22 US US16/691,822 patent/US10888628B2/en active Active
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2022
- 2022-01-27 JP JP2022011052A patent/JP2022062121A/ja active Pending
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2023
- 2023-08-08 US US18/446,243 patent/US20240000974A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
PE20181922A1 (es) | 2018-12-11 |
CN109562191A (zh) | 2019-04-02 |
US20190343966A1 (en) | 2019-11-14 |
EP3452102A1 (en) | 2019-03-13 |
SG10202009849WA (en) | 2020-11-27 |
US10888628B2 (en) | 2021-01-12 |
US20240000974A1 (en) | 2024-01-04 |
RU2018136611A3 (pt) | 2020-08-11 |
JP2022062121A (ja) | 2022-04-19 |
TWI791433B (zh) | 2023-02-11 |
CA3019425A1 (en) | 2017-10-19 |
AU2017248659A1 (en) | 2018-10-18 |
EP3452102B1 (en) | 2024-06-12 |
JP7347933B2 (ja) | 2023-09-20 |
US11779656B2 (en) | 2023-10-10 |
JP2019513794A (ja) | 2019-05-30 |
IL262215B2 (en) | 2023-07-01 |
TW201741458A (zh) | 2017-12-01 |
WO2017180857A1 (en) | 2017-10-19 |
IL262215B1 (en) | 2023-03-01 |
US20200085975A1 (en) | 2020-03-19 |
KR20190032274A (ko) | 2019-03-27 |
MX2018012537A (es) | 2019-02-25 |
RU2762257C2 (ru) | 2021-12-17 |
RU2018136611A (ru) | 2020-05-15 |
AU2017248659B2 (en) | 2022-08-11 |
SG11201808422QA (en) | 2018-10-30 |
IL262215A (en) | 2018-11-29 |
SA518400233B1 (ar) | 2022-08-23 |
KR102450833B1 (ko) | 2022-10-05 |
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