AU2016232146B2 - Optimized liver-specific expression systems for FVIII and FIX - Google Patents

Optimized liver-specific expression systems for FVIII and FIX Download PDF

Info

Publication number
AU2016232146B2
AU2016232146B2 AU2016232146A AU2016232146A AU2016232146B2 AU 2016232146 B2 AU2016232146 B2 AU 2016232146B2 AU 2016232146 A AU2016232146 A AU 2016232146A AU 2016232146 A AU2016232146 A AU 2016232146A AU 2016232146 B2 AU2016232146 B2 AU 2016232146B2
Authority
AU
Australia
Prior art keywords
seq
promoter
nucleic acid
vector
mvm
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.)
Ceased
Application number
AU2016232146A
Other languages
English (en)
Other versions
AU2016232146A1 (en
Inventor
Marinee Chuah
Thierry Vandendriessche
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.)
Vrije Universiteit Brussel
Original Assignee
Vrije Universiteit Brussel
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 Vrije Universiteit Brussel filed Critical Vrije Universiteit Brussel
Publication of AU2016232146A1 publication Critical patent/AU2016232146A1/en
Application granted granted Critical
Publication of AU2016232146B2 publication Critical patent/AU2016232146B2/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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)
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • C12N2750/14111—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
    • C12N2750/14311—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
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • C12N2830/36—Vector systems having a special element relevant for transcription being a transcription termination element
    • 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
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2016232146A 2015-03-17 2016-03-17 Optimized liver-specific expression systems for FVIII and FIX Ceased AU2016232146B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15159395.1 2015-03-17
EP15159395 2015-03-17
PCT/EP2016/055825 WO2016146757A1 (en) 2015-03-17 2016-03-17 Optimized liver-specific expression systems for fviii and fix

Publications (2)

Publication Number Publication Date
AU2016232146A1 AU2016232146A1 (en) 2017-10-12
AU2016232146B2 true AU2016232146B2 (en) 2021-11-04

Family

ID=52686196

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016232146A Ceased AU2016232146B2 (en) 2015-03-17 2016-03-17 Optimized liver-specific expression systems for FVIII and FIX

Country Status (8)

Country Link
US (2) US11007280B2 (enExample)
EP (1) EP3270944B1 (enExample)
JP (1) JP6879486B2 (enExample)
AU (1) AU2016232146B2 (enExample)
CA (1) CA2979495A1 (enExample)
DK (1) DK3270944T3 (enExample)
ES (1) ES2764453T3 (enExample)
WO (1) WO2016146757A1 (enExample)

Families Citing this family (43)

* 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
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
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
KR102404550B1 (ko) 2015-11-13 2022-05-31 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
WO2018178067A1 (en) 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
JP2020519272A (ja) 2017-05-09 2020-07-02 エモリー ユニバーシティー 凝固因子変異体及びその使用
TWI753168B (zh) * 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體
US11452749B2 (en) * 2017-07-10 2022-09-27 Uniqure Ip B.V. Means and methods for AAV gene therapy in humans
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
EP3799569A1 (en) * 2018-07-27 2021-04-07 Zentrum für Forschungsförderung in der Pädiatrie GmbH Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles
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
IL326835A (en) 2018-10-16 2026-04-01 Blueallele Llc Methods for targeted insertion of dna genes
JP2022505876A (ja) * 2018-10-26 2022-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセル 遺伝子治療を向上させる新しいツール及びその使用
JP7529286B2 (ja) * 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用
CA3118936A1 (en) * 2018-11-16 2020-05-22 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
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
WO2020160303A1 (en) 2019-02-01 2020-08-06 Baxalta Incorporated Gene therapy of hemophilia b using viral vectors encoding recombinant fix variants with increased expression
GB201907493D0 (en) 2019-05-28 2019-07-10 Ospedale San Raffaele Agents and methods for treating viral infections
US20230038502A1 (en) * 2019-06-11 2023-02-09 Shire Human Genetic Therapies, Inc. Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
WO2021081280A1 (en) 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
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
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
CA3173696A1 (en) 2020-07-17 2022-01-20 Volker Sandig Hyperactive transposons and transposases
MX2023003019A (es) * 2020-09-16 2023-05-08 Generation Bio Co Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah).
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
GB202018657D0 (en) 2020-11-26 2021-01-13 Ospedale San Raffaele Srl Agents and methods for increasing liver immune response
CN112608921B (zh) * 2020-12-23 2022-04-08 武汉枢密脑科学技术有限公司 用于肝脏中蛋白质表达的重组启动子、载体及其应用
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途
CN117377771A (zh) * 2021-05-12 2024-01-09 特莱索恩基金会 载体系统
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
EP4402267A4 (en) * 2021-09-16 2025-11-26 Generation Bio Co Liver-specific expression cassettes, vectors, and their uses for expressing therapeutic proteins
KR20230166039A (ko) 2022-05-27 2023-12-06 주식회사 엔세이지 항응고인자 9 분비 유도만능줄기세포를 이용한 b형 혈우병 치료제 개발
WO2025008774A1 (en) * 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
WO2025239324A1 (ja) * 2024-05-13 2025-11-20 中外製薬株式会社 遺伝子改変非ヒト動物

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007149852A2 (en) * 2006-06-19 2007-12-27 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
WO2009130208A1 (en) * 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8071740B2 (en) * 2000-11-17 2011-12-06 Vascular Biogenics Ltd. Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
US20040185555A1 (en) * 2003-03-17 2004-09-23 Emini Emilio A. Adenovirus serotype 24 vectors, nucleic acids and virus produced thereby
US8008468B2 (en) * 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
SI1986661T1 (sl) * 2006-02-08 2018-12-31 Genzyme Corporation Genska terapija za Niemann-Pickovo bolezen tipa A
WO2007120533A2 (en) * 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2014063753A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007149852A2 (en) * 2006-06-19 2007-12-27 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
WO2009130208A1 (en) * 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof

Also Published As

Publication number Publication date
AU2016232146A1 (en) 2017-10-12
DK3270944T3 (da) 2020-01-27
WO2016146757A1 (en) 2016-09-22
CA2979495A1 (en) 2016-09-22
EP3270944B1 (en) 2019-10-23
EP3270944A1 (en) 2018-01-24
ES2764453T3 (es) 2020-06-03
US20210369868A1 (en) 2021-12-02
US11007280B2 (en) 2021-05-18
US20180071406A1 (en) 2018-03-15
JP6879486B2 (ja) 2021-06-02
JP2018513678A (ja) 2018-05-31

Similar Documents

Publication Publication Date Title
AU2018306307B2 (en) Enhanced chimeric antigen receptors and use thereof
KR102729768B1 (ko) 트랜스제닉 선택 방법 및 조성물
AU2016229000B2 (en) Recombinant Glut1 adeno-associated viral vector constructs and related methods for restoring Glut1 expression
AU774643B2 (en) Compositions and methods for use in recombinational cloning of nucleic acids
AU2017323629B9 (en) PD-1 homing endonuclease variants, compositions, and methods of use
AU2019257708B2 (en) Expression of human FOXP3 in gene edited T cells
CN112912112B (zh) 肝特异性核酸调节元件以及其方法及用途
AU2024202827A1 (en) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
DK2768848T3 (en) METHODS AND PROCEDURES FOR EXPRESSION AND SECRETARY OF PEPTIDES AND PROTEINS
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
TW202317753A (zh) 經武裝之嵌合受體及其使用方法
CN110856724B (zh) 包含核酸及car修饰的免疫细胞的治疗剂及其应用
CN115768890A (zh) 通过分子和物理启动对t细胞免疫疗法的热控制
KR20220041214A (ko) Il-1 수퍼패밀리의 사이토카인의 시공적 제한 활성으로 무장된 면역반응성 세포
CN115867295A (zh) 用于靶向hpv感染细胞的组合物和方法
KR20230069157A (ko) GJB2를 코딩하는 재조합 아데노 연관 바이러스 (rAAV) 및 그의 용도
CN117795086A (zh) 用于枫糖尿症(msud)中bcaa调节的基因疗法
KR20220142502A (ko) 근육 특이적 핵산 조절 요소 및 이의 방법 및 용도
CN107988259B (zh) SmartBac杆状病毒表达系统及其应用
AU2018326582B2 (en) Retinal promoter and uses thereof
KR102712198B1 (ko) 재조합 발생이 최소화된 유전자치료 벡터, 상기 벡터를 포함하는 재조합 레트로바이러스 및 상기 재조합 레트로바이러스를 포함하는 암의 예방 또는 치료용 약학 조성물
HK40082332B (en) Tools and methods for using cell division loci to control proliferation of cells
HK40082332A (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法
HK40041692A (en) Expression of human foxp3 in gene edited t cells
PL228024B1 (pl) Zestaw wektorów ekspresyjnych

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired