CN108368164A - CpG减少的因子VIII变体、组合物和方法以及用于治疗止血障碍的用途 - Google Patents

CpG减少的因子VIII变体、组合物和方法以及用于治疗止血障碍的用途 Download PDF

Info

Publication number
CN108368164A
CN108368164A CN201680063183.9A CN201680063183A CN108368164A CN 108368164 A CN108368164 A CN 108368164A CN 201680063183 A CN201680063183 A CN 201680063183A CN 108368164 A CN108368164 A CN 108368164A
Authority
CN
China
Prior art keywords
aav
nucleic acid
vector
fviii
acid variant
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.)
Pending
Application number
CN201680063183.9A
Other languages
English (en)
Chinese (zh)
Inventor
泽维尔·安谷拉
萨姆·贤-艾·沈
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Star Fire Treatment Co Ltd
Original Assignee
Star Fire Treatment Co Ltd
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 Star Fire Treatment Co Ltd filed Critical Star Fire Treatment Co Ltd
Publication of CN108368164A publication Critical patent/CN108368164A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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)
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10342—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Transplantation (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CN201680063183.9A 2015-10-30 2016-10-31 CpG减少的因子VIII变体、组合物和方法以及用于治疗止血障碍的用途 Pending CN108368164A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201562249001P 2015-10-30 2015-10-30
US62/249,001 2015-10-30
US201662331872P 2016-05-04 2016-05-04
US62/331,872 2016-05-04
US201662349532P 2016-06-13 2016-06-13
US62/349,532 2016-06-13
US201662357874P 2016-07-01 2016-07-01
US62/357,874 2016-07-01
PCT/US2016/059793 WO2017075619A1 (en) 2015-10-30 2016-10-31 Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Publications (1)

Publication Number Publication Date
CN108368164A true CN108368164A (zh) 2018-08-03

Family

ID=58630834

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680063183.9A Pending CN108368164A (zh) 2015-10-30 2016-10-31 CpG减少的因子VIII变体、组合物和方法以及用于治疗止血障碍的用途

Country Status (19)

Country Link
US (3) US20180312571A1 (https=)
EP (1) EP3368560B1 (https=)
JP (3) JP7088831B2 (https=)
KR (1) KR20180090795A (https=)
CN (1) CN108368164A (https=)
AU (1) AU2016344030B2 (https=)
BR (1) BR112018008766A2 (https=)
CA (1) CA3002689A1 (https=)
CL (1) CL2018001107A1 (https=)
CO (1) CO2018005306A2 (https=)
IL (1) IL258959B2 (https=)
MX (2) MX2018005490A (https=)
MY (1) MY190926A (https=)
PE (2) PE20231949A1 (https=)
PH (1) PH12018501055A1 (https=)
RU (1) RU2745506C2 (https=)
SA (1) SA518391463B1 (https=)
SG (1) SG11201802972UA (https=)
WO (1) WO2017075619A1 (https=)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113226352A (zh) * 2018-08-24 2021-08-06 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
CN113543814A (zh) * 2019-01-18 2021-10-22 布里斯托尔大学 用于治疗肾病综合征的aav基因疗法
CN113874513A (zh) * 2019-03-13 2021-12-31 世代生物公司 非病毒dna载体及其用于表达fviii治疗剂的用途
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
US12121593B2 (en) 2020-06-15 2024-10-22 The University Of Bristol Therapy
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

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
PE20200722A1 (es) * 2017-08-01 2020-07-21 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii)
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
TW202016298A (zh) 2018-05-14 2020-05-01 美商拜奧馬林製藥公司 Aav 載體於幼年受試者中之穩定表現
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
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
CA3119349A1 (en) * 2018-11-16 2020-05-22 Spark Therapeutics, Inc. In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies
SG11202108357PA (en) 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
CN114929735A (zh) * 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
KR20250007615A (ko) * 2022-04-28 2025-01-14 조인트 스탁 컴퍼니 "바이오케드" Fviii-bdd를 인코딩하는 코돈 최적화된 핵산
JP2025529195A (ja) * 2022-08-31 2025-09-04 ハイドロジーン セラピューティクス，インコーポレイテッド 流体力学的遺伝子送達のための方法および組成物
WO2025164792A1 (ja) * 2024-02-02 2025-08-07 学校法人自治医科大学 高比活性／高分泌発現型の第ｖｉｉｉ因子改変体、高発現型の第ｖｉｉｉ因子コード核酸及びそれらの用途

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101035899A (zh) * 2004-08-03 2007-09-12 基因技术股份公司 通过改变CpG含量来调节基因表达的方法
WO2015013313A2 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
WO2015121501A1 (en) * 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
US20150273082A1 (en) * 2009-07-08 2015-10-01 Ucl Business Plc Optimised coding sequence and promoter
WO2016025764A2 (en) * 2014-08-13 2016-02-18 The Children's Hospital Of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6093699A (en) 1987-07-09 2000-07-25 The University Of Manitoba Method for gene therapy involving suppression of an immune response
WO1994017810A1 (en) 1993-02-12 1994-08-18 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
EP0708658A4 (en) 1993-04-16 1997-05-21 Wistar Inst RECOMBINANT CYTOMEGALOVIRUS VACCINE
WO1995005864A1 (en) 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
AT401270B (de) 1994-09-26 1996-07-25 Immuno Ag Verfahren zur quantifizierung von genomischer dna
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
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
EA001616B1 (ru) 1995-02-28 2001-06-25 Зе Риджентс Оф Зи Юнивесити Оф Кэлифоньэ Способ лечения болезни сердца, способ лечения недостаточности периферических сосудов и способ ограничения доставки и экспрессии трансгенной конструкции в определенном органе или структуре
AU2319497A (en) 1996-03-07 1997-09-22 Regents Of The University Of California, The Helper-free, totally defective adenovirus for gene therapy
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
EP1240900B1 (en) * 1999-12-24 2009-05-06 Juridical Foundation, The Chemo-Sero-Therapeutic Research Institute Factor viii or von willebrand factor for the treatment of hemorrhagic diseases associating thrombopathy
PL206105B1 (pl) * 2000-09-19 2010-07-30 Emory Universityemory University Zmodyfikowany ludzki czynnik VIII oraz sposób modyfikacji ludzkiego czynnika VIII ze zmniejszeniem reaktywności na przeciwciało hamujące i zachowaniem aktywności prokoagulacyjnej
GB0908515D0 (en) 2009-05-18 2009-06-24 Apitope Technology Bristol Ltd Peptide
PT3513804T (pt) 2011-07-08 2022-06-02 Bioverativ Therapeutics Inc Polipéptidos fator viii híbridos e quiméricos, e métodos de utilização dos mesmos
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
EP2822577B1 (en) * 2012-02-15 2019-02-06 Bioverativ Therapeutics Inc. Recombinant factor viii proteins
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
WO2015054439A2 (en) * 2013-10-08 2015-04-16 Haplomics, Inc. Hybrid factor viii polypeptides for use to treat hemophilia a
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
PE20200722A1 (es) 2017-08-01 2020-07-21 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii)

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101035899A (zh) * 2004-08-03 2007-09-12 基因技术股份公司 通过改变CpG含量来调节基因表达的方法
US20150273082A1 (en) * 2009-07-08 2015-10-01 Ucl Business Plc Optimised coding sequence and promoter
WO2015013313A2 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
WO2015121501A1 (en) * 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
WO2016025764A2 (en) * 2014-08-13 2016-02-18 The Children's Hospital Of Philadelphia An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SATYA 等: "《Proceedings of the 2003 IEEE Bioinformatics Conference. CSB2003》", 31 December 2003 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113226352A (zh) * 2018-08-24 2021-08-06 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
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
CN113543814A (zh) * 2019-01-18 2021-10-22 布里斯托尔大学 用于治疗肾病综合征的aav基因疗法
US12544460B2 (en) 2019-01-18 2026-02-10 The University Of Bristol AAV gene therapy for treating nephrotic syndrome
CN113874513A (zh) * 2019-03-13 2021-12-31 世代生物公司 非病毒dna载体及其用于表达fviii治疗剂的用途
US12121593B2 (en) 2020-06-15 2024-10-22 The University Of Bristol Therapy
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
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

Also Published As

Publication number Publication date
NZ741860A (en) 2024-12-20
SA518391463B1 (ar) 2022-12-13
EP3368560B1 (en) 2026-03-25
RU2018119710A (ru) 2019-12-06
JP2019503649A (ja) 2019-02-14
PE20231949A1 (es) 2023-12-05
PH12018501055A1 (en) 2019-01-28
IL258959B2 (en) 2024-10-01
SG11201802972UA (en) 2018-05-30
WO2017075619A1 (en) 2017-05-04
EP3368560A4 (en) 2019-03-27
JP2024170416A (ja) 2024-12-10
US20180312571A1 (en) 2018-11-01
CA3002689A1 (en) 2017-05-04
CO2018005306A2 (es) 2018-11-30
MX2023001600A (es) 2023-03-09
MX2018005490A (es) 2018-08-16
RU2018119710A3 (https=) 2020-04-02
EP3368560A1 (en) 2018-09-05
JP2022137029A (ja) 2022-09-21
IL258959A (en) 2018-06-28
KR20180090795A (ko) 2018-08-13
PE20181168A1 (es) 2018-07-19
US20170216408A1 (en) 2017-08-03
AU2016344030A1 (en) 2018-05-10
CL2018001107A1 (es) 2018-10-05
US20250171521A1 (en) 2025-05-29
US11168124B2 (en) 2021-11-09
RU2745506C2 (ru) 2021-03-25
JP7088831B2 (ja) 2022-06-21
IL258959B1 (en) 2024-06-01
AU2016344030B2 (en) 2021-05-06
BR112018008766A2 (en) 2018-10-30
MY190926A (en) 2022-05-20

Similar Documents

Publication Publication Date Title
EP3368560B1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
JP7237903B2 (ja) 止血疾患を治療するための変異因子ｖｉｉｉのゲノム組込及び発現のための改良された発現カセット
US20240131126A1 (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs, and tissues
JP7499174B2 (ja) 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法
US20190144524A1 (en) Factor viii variants, nucleic acid sequences, and methods and uses for treatment of hemostasis disorders
BR112017002781B1 (pt) Variante de ácido nucleico codificando o fator viii, seu uso, vetor de expressão, vetor viral, vetor aav, célula hospedeira in vitro e composição farmacêutica

Legal Events

Date Code Title Description
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination