DK1804839T3 - Forbedret ekspression af faktor ix i genterapivektorer - Google Patents

Forbedret ekspression af faktor ix i genterapivektorer

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Publication number
DK1804839T3
DK1804839T3 DK05796011.4T DK05796011T DK1804839T3 DK 1804839 T3 DK1804839 T3 DK 1804839T3 DK 05796011 T DK05796011 T DK 05796011T DK 1804839 T3 DK1804839 T3 DK 1804839T3
Authority
DK
Denmark
Prior art keywords
factor
vectors
gentherapy
improved expression
expression
Prior art date
Application number
DK05796011.4T
Other languages
English (en)
Inventor
John Trainor Gray
Original Assignee
St Jude Childrens Res Hospital
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 St Jude Childrens Res Hospital filed Critical St Jude Childrens Res Hospital
Application granted granted Critical
Publication of DK1804839T3 publication Critical patent/DK1804839T3/da

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/647Blood coagulation factors not provided for in a preceding group or according to more than one of the proceeding groups
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775Apolipopeptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81Protease inhibitors
    • C07K14/8107Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121Serpins
    • C07K14/8125Alpha-1-antitrypsin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/15Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/20Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C12N2830/205Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian
DK05796011.4T 2004-09-22 2005-09-09 Forbedret ekspression af faktor ix i genterapivektorer DK1804839T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US61213504P 2004-09-22 2004-09-22
PCT/US2005/032078 WO2006036502A2 (en) 2004-09-22 2005-09-09 Improved expression of factor ix in gene therapy vectors

Publications (1)

Publication Number Publication Date
DK1804839T3 true DK1804839T3 (da) 2012-04-10

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
DK05796011.4T DK1804839T3 (da) 2004-09-22 2005-09-09 Forbedret ekspression af faktor ix i genterapivektorer
DK12150318.9T DK2438931T3 (da) 2004-09-22 2005-09-09 Forbedret ekspression af faktor ix i genterapivektorer

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK12150318.9T DK2438931T3 (da) 2004-09-22 2005-09-09 Forbedret ekspression af faktor ix i genterapivektorer

Country Status (6)

Country Link
US (2) US8030065B2 (da)
EP (2) EP2438931B1 (da)
AT (1) ATE549037T1 (da)
DK (2) DK1804839T3 (da)
ES (2) ES2436741T3 (da)
WO (1) WO2006036502A2 (da)

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ATE549037T1 (de) * 2004-09-22 2012-03-15 St Jude Childrens Res Hospital Verbesserte expression von faktor-ix in gentherapie-vektoren
CA2722238C (en) * 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
CA2737094C (en) * 2008-09-15 2018-02-20 Paolo Simioni Factor ix polypeptide mutant, its uses and a method for its production
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BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
JP6735672B2 (ja) 2013-09-12 2020-08-05 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第viii因子ベクター
EP3117005A1 (en) 2014-03-10 2017-01-18 UniQure IP B.V. Further improved aav vectors produced in insect cells
MX2016011690A (es) 2014-03-21 2016-11-07 Univ Leland Stanford Junior Edicion del genoma sin nucleasas.
EP3160478A4 (en) * 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11697825B2 (en) * 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN115094062A (zh) * 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
CA2983593A1 (en) * 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
PE20180675A1 (es) * 2015-06-23 2018-04-19 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos
AU2016326602A1 (en) 2015-09-24 2018-03-29 Biomarin Pharmaceutical Inc. Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
WO2017066579A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
SG10201913278PA (en) 2016-02-01 2020-02-27 Bioverativ Therapeutics Inc Optimized factor viii genes
JP7347933B2 (ja) 2016-04-15 2023-09-20 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 血友病a治療に対する遺伝子療法
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
JP2020505044A (ja) * 2017-01-26 2020-02-20 サンガモ セラピューティクス, インコーポレイテッド B細胞操作
WO2018217731A1 (en) 2017-05-22 2018-11-29 Baxalta Incorporated Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b
CN114875051A (zh) 2017-05-31 2022-08-09 北卡罗来纳大学教堂山分校 优化的人凝血因子ix基因表达盒及其应用
KR20200041310A (ko) * 2017-07-10 2020-04-21 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
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
GB2576508A (en) * 2018-08-20 2020-02-26 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3810770A1 (en) * 2018-08-20 2021-04-28 Ucl Business Ltd Factor ix encoding nucleotides
US20230250412A1 (en) * 2019-01-24 2023-08-10 The Children's Hospital Of Philadelphia Compositions and Methods for Modulating Factor IX Function
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MX2022008415A (es) 2020-01-08 2022-08-08 Obsidian Therapeutics Inc Composiciones y metodos para la regulacion ajustable de la transcripcion.
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CN113817759B (zh) * 2020-07-10 2023-06-02 南京吉迈生物技术有限公司 修饰的因子ix、组合物、方法及其在基因治疗中的应用
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JP2004532039A (ja) * 2001-03-26 2004-10-21 ザ ボード オブ トラスティーズ オブ ザ リーランド スタンフォード ジュニア ユニバーシティ ヘルパー依存性アデノウイルスベクター系およびその系の使用方法
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ATE549037T1 (de) * 2004-09-22 2012-03-15 St Jude Childrens Res Hospital Verbesserte expression von faktor-ix in gentherapie-vektoren

Also Published As

Publication number Publication date
DK2438931T3 (da) 2013-12-02
EP2438931A1 (en) 2012-04-11
WO2006036502A8 (en) 2007-10-04
ATE549037T1 (de) 2012-03-15
EP1804839B1 (en) 2012-03-14
EP1804839A2 (en) 2007-07-11
EP2438931B1 (en) 2013-11-13
ES2436741T3 (es) 2014-01-07
US8030065B2 (en) 2011-10-04
US8168425B2 (en) 2012-05-01
EP1804839A4 (en) 2009-12-09
ES2381796T3 (es) 2012-05-31
WO2006036502A2 (en) 2006-04-06
US20110287532A1 (en) 2011-11-24
US20080153156A1 (en) 2008-06-26

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