KR102450833B1 - 혈우병 a 치료용 유전자 요법 - Google Patents

혈우병 a 치료용 유전자 요법 Download PDF

Info

Publication number
KR102450833B1
KR102450833B1 KR1020187032617A KR20187032617A KR102450833B1 KR 102450833 B1 KR102450833 B1 KR 102450833B1 KR 1020187032617 A KR1020187032617 A KR 1020187032617A KR 20187032617 A KR20187032617 A KR 20187032617A KR 102450833 B1 KR102450833 B1 KR 102450833B1
Authority
KR
South Korea
Prior art keywords
leu
gly
ser
vector
pro
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
KR1020187032617A
Other languages
English (en)
Korean (ko)
Other versions
KR20190032274A (ko
Inventor
릴리 왕
제임스 엠. 윌슨
제니 아그네스 시드레인
Original Assignee
더 트러스티스 오브 더 유니버시티 오브 펜실베니아
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 filed Critical 더 트러스티스 오브 더 유니버시티 오브 펜실베니아
Publication of KR20190032274A publication Critical patent/KR20190032274A/ko
Application granted granted Critical
Publication of KR102450833B1 publication Critical patent/KR102450833B1/ko
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
KR1020187032617A 2016-04-15 2017-04-13 혈우병 a 치료용 유전자 요법 Expired - Fee Related KR102450833B1 (ko)

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 (2)

Publication Number Publication Date
KR20190032274A KR20190032274A (ko) 2019-03-27
KR102450833B1 true KR102450833B1 (ko) 2022-10-05

Family

ID=59253992

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020187032617A Expired - Fee Related KR102450833B1 (ko) 2016-04-15 2017-04-13 혈우병 a 치료용 유전자 요법

Country Status (17)

Country Link
US (3) US11779656B2 (enExample)
EP (1) EP3452102B9 (enExample)
JP (2) JP7347933B2 (enExample)
KR (1) KR102450833B1 (enExample)
CN (1) CN109562191A (enExample)
AU (1) AU2017248659B2 (enExample)
BR (1) BR112018071200A2 (enExample)
CA (1) CA3019425A1 (enExample)
IL (1) IL262215B2 (enExample)
MX (1) MX2018012537A (enExample)
PE (2) PE20181922A1 (enExample)
RU (1) RU2762257C2 (enExample)
SA (1) SA518400233B1 (enExample)
SG (2) SG10202009849WA (enExample)
TW (1) TWI791433B (enExample)
WO (1) WO2017180857A1 (enExample)
ZA (1) ZA201806477B (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2985035T3 (es) 2008-09-15 2024-11-04 Uniqure Biopharma B V Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción
CN108463211B (zh) 2015-11-16 2022-09-27 全国儿童医院研究所 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法
PE20181922A1 (es) * 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
IL279789B2 (en) 2018-06-29 2026-04-01 Res Inst Nationwide Childrens Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
TW202028225A (zh) * 2018-09-21 2020-08-01 英商夜星克有限公司 用於治療色素性視網膜炎之組合物及方法
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
JP2022514112A (ja) * 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
IL326835A (en) 2018-10-16 2026-04-01 Blueallele Llc Methods for targeted insertion of dna genes
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
KR20210133242A (ko) * 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
CA3150964A1 (en) 2019-08-21 2021-02-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
WO2021084276A2 (en) * 2019-11-01 2021-05-06 Freeline Therapeutics Limited Factor viii construct
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
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
US20230175014A1 (en) * 2020-04-27 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
KR20230042513A (ko) * 2020-07-29 2023-03-28 프리라인 테라퓨틱스 리미티드 폴리펩티드
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
AU2022214529A1 (en) * 2021-02-01 2023-08-03 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of niemann pick type a disease
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
EP4511055A4 (en) * 2022-04-20 2026-04-01 Askbio Inc Adeno-associated virus therapy using a codon-optimized nucleic acid encoding factor VIII
US20250352626A1 (en) * 2022-06-10 2025-11-20 University Of Florida Research Foundation, Incorporated Immune tolerance induction to viral capsids
WO2025180508A1 (en) * 2024-02-29 2025-09-04 Shanghai Vitalgen Biopharma Co., Ltd. Human factor viii variant expression cassettes and uses thereof for treating hemophilia a

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070243168A1 (en) 2006-03-30 2007-10-18 Mark Kay Minigene expression cassette
WO2014064277A1 (en) * 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US20150071883A1 (en) 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Family Cites Families (63)

* Cited by examiner, † Cited by third party
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
WO1998000542A2 (en) * 1996-07-03 1998-01-08 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia
JP2002517180A (ja) 1997-12-05 2002-06-18 ジ・イミユーン・リスポンス・コーポレーシヨン 増大された発現を示す新規ベクターおよび遺伝子
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
WO2001036620A2 (en) 1999-11-16 2001-05-25 Genzyme Corporation Vectors and transgenies with regulatory elements for gene delivery to the liver
DK1266006T3 (da) 2000-03-22 2006-01-16 Octagene Gmbh Fremstilling af rekombinante muteiner af blodkoagulationsfaktor VIII i humane cellelinier
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
DE60230456D1 (de) 2001-10-05 2009-01-29 Expression Therapeutics Llc Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
JP4804356B2 (ja) 2003-10-16 2011-11-02 クラウド ネグリエー 第ＶＩＩＩ因子及びその誘導体の高発現レベルのための改変されたｃＤＮＡ
ATE463572T1 (de) 2003-12-03 2010-04-15 Univ Rochester Rekombinanter faktor viii mit erhöhter spezifischer aktivität
ES2436741T3 (es) 2004-09-22 2014-01-07 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
KR20080007226A (ko) 2005-04-14 2008-01-17 체에스엘 베링 게엠베하 증가된 안정성을 가진 변형된 응고 인자 ⅷ 및 이의 유도체
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
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
CN101511373B (zh) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
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
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
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
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
EP2313531A1 (en) 2008-06-25 2011-04-27 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
AU2010227419A1 (en) 2009-03-27 2011-10-20 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
WO2011109380A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
US9315825B2 (en) * 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 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
WO2012028681A1 (en) * 2010-09-02 2012-03-08 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
EP3513802B1 (en) * 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector 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
KR102554850B1 (ko) * 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
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
CN108884145B (zh) * 2015-11-13 2023-08-22 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
PE20181922A1 (es) * 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070243168A1 (en) 2006-03-30 2007-10-18 Mark Kay Minigene expression cassette
WO2014064277A1 (en) * 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US20150071883A1 (en) 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Blood. 2006. Vol.108. NO.1, pp.107-115.*
Gene Therapy. 2008. Vol.15, pp.289-297.

Also Published As

Publication number Publication date
US10888628B2 (en) 2021-01-12
US20240000974A1 (en) 2024-01-04
AU2017248659B2 (en) 2022-08-11
KR20190032274A (ko) 2019-03-27
IL262215B2 (en) 2023-07-01
CA3019425A1 (en) 2017-10-19
EP3452102B1 (en) 2024-06-12
SA518400233B1 (ar) 2022-08-23
US20200085975A1 (en) 2020-03-19
US20190343966A1 (en) 2019-11-14
BR112018071200A2 (pt) 2019-04-09
ZA201806477B (en) 2024-09-25
IL262215A (en) 2018-11-29
SG10202009849WA (en) 2020-11-27
AU2017248659A1 (en) 2018-10-18
RU2762257C2 (ru) 2021-12-17
PE20181922A1 (es) 2018-12-11
JP2022062121A (ja) 2022-04-19
US11779656B2 (en) 2023-10-10
EP3452102A1 (en) 2019-03-13
WO2017180857A1 (en) 2017-10-19
EP3452102B9 (en) 2024-09-04
RU2018136611A (ru) 2020-05-15
MX2018012537A (es) 2019-02-25
IL262215B1 (en) 2023-03-01
CN109562191A (zh) 2019-04-02
PE20250736A1 (es) 2025-03-10
TW201741458A (zh) 2017-12-01
SG11201808422QA (en) 2018-10-30
JP2019513794A (ja) 2019-05-30
TWI791433B (zh) 2023-02-11
JP7347933B2 (ja) 2023-09-20
RU2018136611A3 (enExample) 2020-08-11

Similar Documents

Publication Publication Date Title
AU2017248659B2 (en) Gene therapy for treating hemophilia A
JP7613759B2 (ja) ハンチントン病のａａｖ処置
AU2019261438B2 (en) Expression of FOXP3 in edited CD34+ cells
DK2718440T3 (en) NUCLEASE ACTIVITY PROTEIN, FUSION PROTEINS AND APPLICATIONS THEREOF
AU2019257708B2 (en) Expression of human FOXP3 in gene edited T cells
CN108753824B (zh) 用于治疗视网膜营养不良的病毒载体
KR101982360B1 (ko) 콤팩트 tale-뉴클레아제의 발생 방법 및 이의 용도
KR20210149060A (ko) Tn7-유사 트랜스포존을 사용한 rna-유도된 dna 통합
AU2014372562B2 (en) A method of making adenovirus and corresponding plasmids
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
AU2016370590B2 (en) Composition for treatment of Crigler-Najjar syndrome
KR20210005179A (ko) 헤모글로빈-관련 돌연변이를 편집하기 위한 상동성-지정 복구 주형 설계 및 전달
CN111733174B (zh) 一种分离的核酸分子及其用途
CN112912112B (zh) 肝特异性核酸调节元件以及其方法及用途
CN113260704B (zh) CYP4V2和RdCVF在制备药物中的用途
CN115362000A (zh) 使用多核苷酸沉默和替换的神经退行性病症的基因疗法
CN112203697A (zh) 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途
CN116323942A (zh) 用于基因组编辑的组合物及其使用方法
CN113897390B (zh) 一种非洲猪瘟的重组腺病毒疫苗及其构建方法
CA2527954A1 (en) Modified fiber proteins for efficient receptor binding
CN116323955A (zh) 通过crispr/cas介导的体内末端解析拯救重组腺病毒
RU2812852C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фактора viii (fviii)
CN115003804A (zh) 基因构建体
HK40044016A (en) Expression of foxp3 in edited cd34+ cells
TW202603176A (zh) 以密碼子最佳化核酸編碼ｌａｍｐ２ｂ的治療性腺相關病毒

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

D13-X000 Search requested

St.27 status event code: A-1-2-D10-D13-srh-X000

D14-X000 Search report completed

St.27 status event code: A-1-2-D10-D14-srh-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

GRNT Written decision to grant
PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

R18-X000 Changes to party contact information recorded

St.27 status event code: A-5-5-R10-R18-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-5-5-R10-R18-oth-X000

PC1903 Unpaid annual fee

St.27 status event code: A-4-4-U10-U13-oth-PC1903

Not in force date: 20250930

Payment event data comment text: Termination Category : DEFAULT_OF_REGISTRATION_FEE

H13 Ip right lapsed

Free format text: ST27 STATUS EVENT CODE: N-4-6-H10-H13-OTH-PC1903 (AS PROVIDED BY THE NATIONAL OFFICE); TERMINATION CATEGORY : DEFAULT_OF_REGISTRATION_FEE

Effective date: 20250930

PC1903 Unpaid annual fee

St.27 status event code: N-4-6-H10-H13-oth-PC1903

Ip right cessation event data comment text: Termination Category : DEFAULT_OF_REGISTRATION_FEE

Not in force date: 20250930