DK2911687T3 - Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf - Google Patents

Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf Download PDF

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Publication number
DK2911687T3
DK2911687T3 DK13789223.8T DK13789223T DK2911687T3 DK 2911687 T3 DK2911687 T3 DK 2911687T3 DK 13789223 T DK13789223 T DK 13789223T DK 2911687 T3 DK2911687 T3 DK 2911687T3
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Prior art keywords
hemophilia
liver
vector
methods
gene therapy
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DK13789223.8T
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Danish (da)
English (en)
Inventor
Thierry Vandendriessche
Marinee Chuah
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Univ Brussel Vrije
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Priority claimed from PCT/EP2012/071297 external-priority patent/WO2014063753A1/en
Application filed by Univ Brussel Vrije filed Critical Univ Brussel Vrije
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Publication of DK2911687T3 publication Critical patent/DK2911687T3/da

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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
    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
DK13789223.8T 2012-10-26 2013-10-25 Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf DK2911687T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
PCT/EP2012/071297 WO2014063753A1 (en) 2012-10-26 2012-10-26 Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
EP2013064054 2013-07-03
PCT/EP2013/072450 WO2014064277A1 (en) 2012-10-26 2013-10-25 Vector for liver-directed gene therapy of hemophilia and methods and use thereof

Publications (1)

Publication Number Publication Date
DK2911687T3 true DK2911687T3 (da) 2019-05-13

Family

ID=49554217

Family Applications (1)

Application Number Title Priority Date Filing Date
DK13789223.8T DK2911687T3 (da) 2012-10-26 2013-10-25 Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf

Country Status (7)

Country Link
US (2) US11419950B2 (enExample)
EP (1) EP2911687B1 (enExample)
JP (1) JP6454643B2 (enExample)
AU (1) AU2013336601B2 (enExample)
CA (1) CA2888931C (enExample)
DK (1) DK2911687T3 (enExample)
WO (1) WO2014064277A1 (enExample)

Families Citing this family (53)

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US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
AU2013344375B2 (en) 2012-11-16 2017-09-14 Transposagen Biopharmaceuticals, Inc. Site-specific enzymes and methods of use
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JP2017518082A (ja) * 2014-06-17 2017-07-06 ポセイダ セラピューティクス， インコーポレイテッド ゲノム中の特異的遺伝子座にタンパク質を指向させるための方法およびその使用
CN107427557B (zh) * 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016127057A1 (en) 2015-02-06 2016-08-11 The University Of North Carolina At Chapel Hill Optimized human clotting factor viii gene expression cassettes and their use
WO2016146757A1 (en) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3283126B1 (en) 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
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WO2016183422A1 (en) 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
ES2886599T3 (es) 2015-06-17 2021-12-20 Poseida Therapeutics Inc Composiciones y métodos para dirigir proteínas a loci específicos en el genoma
PE20181338A1 (es) * 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
AU2016338565B2 (en) 2015-10-14 2021-11-18 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
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US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
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EP3600445A1 (en) 2017-03-27 2020-02-05 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
EP3622065A1 (en) 2017-05-09 2020-03-18 Emory University Clotting factor variants and their use
KR102386890B1 (ko) * 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
KR20240170979A (ko) * 2017-07-10 2024-12-05 유니큐어 아이피 비.브이. 인간에서의 ａａｖ 유전자 요법을 위한 수단 및 방법
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
KR20210034013A (ko) 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
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GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
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EP3870215A1 (en) * 2018-10-26 2021-09-01 Vrije Universiteit Brussel New tools for improving gene therapy and use thereof
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
US11596671B2 (en) 2019-02-01 2023-03-07 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
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TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
MX2023003021A (es) * 2020-09-16 2023-05-24 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con fviii.
CN111926041B (zh) * 2020-10-16 2020-12-25 广州吉妮欧生物科技有限公司 一种SARS-CoV-2假病毒小鼠体内包装系统及其制备方法
WO2022187671A1 (en) * 2021-03-04 2022-09-09 Poseida Therapeutics, Inc. Compositions and methods for the treatment of hemophilia
WO2022204535A1 (en) * 2021-03-25 2022-09-29 The Johns Hopkins University Hydrodynamic gene delivery
US20240269241A1 (en) 2021-06-14 2024-08-15 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
TW202517669A (zh) 2023-07-05 2025-05-01 日商武田藥品工業股份有限公司 用於Ａ型血友病之基因療法的編碼表現增加之重組ＦＶｌｌｌ變異體的病毒載體
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US20190240350A1 (en) 2019-08-08
JP2016500519A (ja) 2016-01-14
JP6454643B2 (ja) 2019-01-16
CA2888931C (en) 2023-09-05
US20230022390A1 (en) 2023-01-26
AU2013336601B2 (en) 2018-01-25
EP2911687B1 (en) 2019-02-13
US11419950B2 (en) 2022-08-23
CA2888931A1 (en) 2014-05-01
AU2013336601A1 (en) 2015-05-28
WO2014064277A1 (en) 2014-05-01
EP2911687A1 (en) 2015-09-02

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