DK1804839T3 - Forbedret ekspression af faktor ix i genterapivektorer - Google Patents
Forbedret ekspression af faktor ix i genterapivektorerInfo
- 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
Links
Classifications
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- 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)
- C12N9/647—Blood coagulation factors not provided for in a preceding group or according to more than one of the proceeding groups
-
- 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
-
- 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
- C07K14/775—Apolipopeptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
- C07K14/8125—Alpha-1-antitrypsin
-
- 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
-
- 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
-
- 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
- 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
- C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
- C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
-
- 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
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
ID=36119370
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) |
Families Citing this family (37)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
PL3889173T3 (pl) | 2013-02-15 | 2023-12-11 | Bioverativ Therapeutics Inc. | Zoptymalizowany gen czynnika viii |
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 |
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 |
MX2022008415A (es) | 2020-01-08 | 2022-08-08 | Obsidian Therapeutics Inc | Composiciones y metodos para la regulacion ajustable de la transcripcion. |
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 |
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 |
EP4079860A1 (en) | 2021-04-23 | 2022-10-26 | uniQure biopharma B.V. | Methods and means for the prevention and/or treatment of joint damage in hemophilia |
WO2023152752A1 (en) * | 2022-02-14 | 2023-08-17 | Ramot At Tel-Aviv University Ltd. | Genetic engineering of b cells and uses thereof in antigen-induced therapeutic protein secretion |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
WO1994029471A1 (en) | 1993-06-10 | 1994-12-22 | Genetic Therapy, Inc. | Adenoviral vectors for treatment of hemophilia |
WO1998041240A1 (en) | 1997-03-14 | 1998-09-24 | The Children's Hospital Of Philadelphia | Methods and compositions for use in gene therapy for treatment of hemophilia |
WO2002064799A2 (en) | 1999-09-28 | 2002-08-22 | Transkaryotic Therapies, Inc. | Optimized messenger rna |
JP4987205B2 (ja) * | 2000-03-03 | 2012-07-25 | ジェネトロニクス, インコーポレイテッド | 遺伝子送達用核酸製剤および使用方法 |
BRPI0109494B8 (pt) | 2000-03-22 | 2021-05-25 | Octagene Gmbh | muteína de fator viii, sequência de dna, vetor, processo para produção da muteína de fator viii, composição farmacêutica e uso da muteína do fator viii |
DE60117550T2 (de) * | 2000-06-01 | 2006-12-07 | University Of North Carolina At Chapel Hill | Doppelsträngige parvovirus-vektoren |
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 |
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 |
ATE437221T1 (de) | 2001-05-14 | 2009-08-15 | Gbp Ip Llc | Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie |
US7232670B2 (en) | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
US20040009151A1 (en) | 2002-04-04 | 2004-01-15 | Kay Mark A. | Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal |
ATE549037T1 (de) * | 2004-09-22 | 2012-03-15 | St Jude Childrens Res Hospital | Verbesserte expression von faktor-ix in gentherapie-vektoren |
-
2005
- 2005-09-09 AT AT05796011T patent/ATE549037T1/de active
- 2005-09-09 EP EP12150318.9A patent/EP2438931B1/en active Active
- 2005-09-09 US US11/575,669 patent/US8030065B2/en active Active
- 2005-09-09 ES ES12150318.9T patent/ES2436741T3/es active Active
- 2005-09-09 EP EP05796011A patent/EP1804839B1/en active Active
- 2005-09-09 DK DK05796011.4T patent/DK1804839T3/da active
- 2005-09-09 DK DK12150318.9T patent/DK2438931T3/da active
- 2005-09-09 ES ES05796011T patent/ES2381796T3/es active Active
- 2005-09-09 WO PCT/US2005/032078 patent/WO2006036502A2/en active Application Filing
-
2011
- 2011-08-04 US US13/198,328 patent/US8168425B2/en active Active
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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