DE69841982D1 - Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie - Google Patents
Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilieInfo
- Publication number
- DE69841982D1 DE69841982D1 DE69841982T DE69841982T DE69841982D1 DE 69841982 D1 DE69841982 D1 DE 69841982D1 DE 69841982 T DE69841982 T DE 69841982T DE 69841982 T DE69841982 T DE 69841982T DE 69841982 D1 DE69841982 D1 DE 69841982D1
- Authority
- DE
- Germany
- Prior art keywords
- compositions
- hemophilia
- treatment
- gene therapy
- associated virus
- 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.)
- Expired - Lifetime
Links
Classifications
-
- 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
- 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
- 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
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US4071197P | 1997-03-14 | 1997-03-14 | |
PCT/US1998/004790 WO1998041240A1 (en) | 1997-03-14 | 1998-03-12 | Methods and compositions for use in gene therapy for treatment of hemophilia |
Publications (1)
Publication Number | Publication Date |
---|---|
DE69841982D1 true DE69841982D1 (de) | 2010-12-16 |
Family
ID=21912503
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DE69841982T Expired - Lifetime DE69841982D1 (de) | 1997-03-14 | 1998-03-12 | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
Country Status (9)
Country | Link |
---|---|
US (3) | US6093392A (de) |
EP (1) | EP1005376B1 (de) |
JP (1) | JP4289687B2 (de) |
AT (1) | ATE486614T1 (de) |
AU (1) | AU745386B2 (de) |
CA (1) | CA2283639A1 (de) |
DE (1) | DE69841982D1 (de) |
ES (1) | ES2355588T3 (de) |
WO (1) | WO1998041240A1 (de) |
Families Citing this family (55)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6531455B1 (en) * | 1995-03-24 | 2003-03-11 | The Regents Of The University Of California | Delivery of polynucleotides by secretory gland expression |
US5885971A (en) * | 1995-03-24 | 1999-03-23 | The Regents Of The University Of California | Gene therapy by secretory gland expression |
US20020037867A1 (en) * | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
US20030215422A1 (en) | 1996-09-11 | 2003-11-20 | John A. Chiorini | Aav4 vector and uses thereof |
US6797505B2 (en) * | 1998-05-27 | 2004-09-28 | Cell Genesys, Inc. | Recombinant AAV vectors for gene therapy of hemophilia A |
US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
WO1999061601A2 (en) * | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
US6759237B1 (en) * | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
ATE374251T1 (de) * | 1999-02-19 | 2007-10-15 | Engene Inc | Zusammensetzungen zur gentherapie von diabetes |
AU5470900A (en) | 1999-06-08 | 2000-12-28 | Children's Hospital Of Philadelphia, The | Methods for preventing formation of inhibitory antibodies in the setting of genetherapy |
US6610906B1 (en) * | 1999-06-09 | 2003-08-26 | The Regents Of The University Of Michigan | Nucleotide sequences for gene regulation and methods of use thereof |
US7217699B2 (en) * | 2000-01-03 | 2007-05-15 | Panagenic International, Inc. | Compositions comprising genome segments and methods of using the same |
US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
ATE438414T1 (de) * | 2000-06-01 | 2009-08-15 | Univ North Carolina | Verfahren und zusammensetzungen zur kontrollierter abgabe von rekombinant parvovirus vektoren |
AU2001268373A1 (en) * | 2000-06-13 | 2001-12-24 | The Children's Hospital Of Philadelphia | Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus |
AU2002226028A1 (en) * | 2000-11-14 | 2002-05-27 | Board Of Regents, Unversity Of Texas Systems | Mutant human factor ix with an increased resistance to inhibition by heparin |
US6794376B2 (en) * | 2000-12-06 | 2004-09-21 | William Beaumont Hospital | Methods and compositions for enhancing diffusion of therapeutic agents through tissue |
US20020159978A1 (en) * | 2001-02-06 | 2002-10-31 | James Allen | Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions |
US7220718B2 (en) | 2001-08-03 | 2007-05-22 | United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Oral treatment of hemophilia |
WO2003018611A2 (en) | 2001-08-24 | 2003-03-06 | University Of Victoria Innovation And Development Corporation | Proaerolysin containing protease activation sequences and methods of use for treatment of prostate cancer |
US20030198620A1 (en) * | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
US20040092008A1 (en) * | 2002-11-12 | 2004-05-13 | Snyder Richard O. | Recombinant AAV vectors for gene therapy of hemophilia A |
US8927269B2 (en) | 2003-05-19 | 2015-01-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Avian adenoassociated virus and uses thereof |
ES2629380T3 (es) | 2003-06-19 | 2017-08-09 | Genzyme Corporation | Viriones de AAV con inmunorreactividad reducida y usos de los mismos |
WO2005056807A2 (en) | 2003-12-04 | 2005-06-23 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health | Bovine adeno-associated viral (baav) vector and uses thereof |
US20060078542A1 (en) * | 2004-02-10 | 2006-04-13 | Mah Cathryn S | Gel-based delivery of recombinant adeno-associated virus vectors |
US7722596B2 (en) * | 2004-02-26 | 2010-05-25 | Osprey Medical, Inc. | Regional cardiac tissue treatment |
ATE549037T1 (de) | 2004-09-22 | 2012-03-15 | St Jude Childrens Res Hospital | Verbesserte expression von faktor-ix in gentherapie-vektoren |
CN101124328A (zh) | 2004-12-15 | 2008-02-13 | 北卡罗来纳查佩尔山大学 | 嵌合载体 |
WO2006089340A2 (en) * | 2005-02-23 | 2006-08-31 | V-Kardia Pty Ltd | Polynucleotide delivery to cardiac tissue |
WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
US8152786B2 (en) * | 2006-11-07 | 2012-04-10 | Osprey Medical, Inc. | Collection catheter and kit |
US9050400B2 (en) * | 2008-08-12 | 2015-06-09 | Osprey Medical, Inc. | Remote sensing catheter system and methods |
US20100041984A1 (en) * | 2008-08-12 | 2010-02-18 | James Edward Shapland | Impedance sensing device and catheter system |
US8889641B2 (en) | 2009-02-11 | 2014-11-18 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
FR2952374B1 (fr) * | 2009-11-06 | 2012-11-16 | Univ Claude Bernard Lyon | Production de facteur ix recombinant dans une lignee cellulaire hepatocytaire humaine |
WO2011057243A2 (en) * | 2009-11-09 | 2011-05-12 | University Of Central Florida Research Foundation, Inc. | Administration of plant expressed oral tolerance agents |
WO2011071988A1 (en) * | 2009-12-08 | 2011-06-16 | Health Research, Inc. | Methods and compositions using peroxiredoxin 1 (prx1) as an adjuvant |
US9295816B2 (en) * | 2009-12-09 | 2016-03-29 | Osprey Medical, Inc. | Catheter with distal and proximal ports |
ES2680915T3 (es) | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
US9175280B2 (en) * | 2010-10-12 | 2015-11-03 | Sangamo Biosciences, Inc. | Methods and compositions for treating hemophilia B |
EP4043025A1 (de) | 2011-06-08 | 2022-08-17 | Translate Bio, Inc. | Lipidnanoteilchenzusammensetzungen und verfahren zur mrna-freisetzung |
EP2721151B1 (de) | 2011-06-20 | 2017-12-06 | Mount Sinai School Of Medicine | Anti-tnf-therapie für mucopolysaccharidosen und andere lysosomale störungen |
US9211372B2 (en) | 2011-08-11 | 2015-12-15 | Osprey Medical, Inc. | Systems and methods for limb treatment |
RU2705249C2 (ru) * | 2013-07-12 | 2019-11-06 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Вектор aav и анализ на нейтрализующие антитела против aav (аденоассоциированного вируса) |
KR102417915B1 (ko) | 2014-03-21 | 2022-07-07 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | 뉴클레아제 없는 게놈 편집 |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
EP4215605A1 (de) * | 2015-12-11 | 2023-07-26 | The Trustees of The University of Pennsylvania | Skalierbares reinigungsverfahren für aav8 |
EA201892347A1 (ru) | 2016-04-15 | 2019-04-30 | Баксалта Инкорпорейтед | Способ и устройство для предоставления фармакокинетической схемы дозирования лекарственных препаратов |
US10896749B2 (en) | 2017-01-27 | 2021-01-19 | Shire Human Genetic Therapies, Inc. | Drug monitoring tool |
US10842853B2 (en) | 2017-05-22 | 2020-11-24 | Baxalta Incorporated | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B |
WO2019083171A2 (ko) | 2017-10-26 | 2019-05-02 | 주식회사 큐로진생명과학 | 솔루블 vegfr-1 변이체 cdna를 함유하는 raav를 포함하는 황반변성 치료용 조성물 |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP0107278B1 (de) * | 1982-08-04 | 1989-11-15 | National Research Development Corporation | Molekulare Klonierung des Gens für menschlichen antihaemophilischen Faktor IX |
US5139941A (en) * | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
NZ244306A (en) * | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
US5834266A (en) | 1993-02-12 | 1998-11-10 | President & Fellows Of Harvard College | Regulated apoptosis |
US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
US5843742A (en) * | 1994-12-16 | 1998-12-01 | Avigen Incorporated | Adeno-associated derived vector systems for gene delivery and integration into target cells |
US5834306A (en) | 1994-12-23 | 1998-11-10 | Sri International | Tissue specific hypoxia regulated therapeutic constructs |
US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
US5817784A (en) | 1996-08-09 | 1998-10-06 | Amgen Inc. | Neurogene |
US5866552A (en) * | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
KR20000068501A (ko) * | 1996-09-06 | 2000-11-25 | 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 재조합 아데노-관련 바이러스 지정 유전자 요법을 위한 방법 |
AU743335B2 (en) * | 1996-09-25 | 2002-01-24 | Cell Genesys, Inc. | Non-invasive administration of adeno-associated viral vectors |
-
1998
- 1998-03-12 WO PCT/US1998/004790 patent/WO1998041240A1/en active IP Right Grant
- 1998-03-12 EP EP98910320A patent/EP1005376B1/de not_active Expired - Lifetime
- 1998-03-12 CA CA002283639A patent/CA2283639A1/en not_active Abandoned
- 1998-03-12 ES ES98910320T patent/ES2355588T3/es not_active Expired - Lifetime
- 1998-03-12 AU AU64586/98A patent/AU745386B2/en not_active Expired
- 1998-03-12 DE DE69841982T patent/DE69841982D1/de not_active Expired - Lifetime
- 1998-03-12 AT AT98910320T patent/ATE486614T1/de not_active IP Right Cessation
- 1998-03-12 US US09/038,910 patent/US6093392A/en not_active Expired - Lifetime
- 1998-03-12 JP JP54061098A patent/JP4289687B2/ja not_active Expired - Lifetime
-
2007
- 2007-10-31 US US11/932,017 patent/US7998734B2/en not_active Expired - Fee Related
-
2011
- 2011-07-08 US US13/178,992 patent/US20110263690A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
JP2001517221A (ja) | 2001-10-02 |
WO1998041240A1 (en) | 1998-09-24 |
US20110263690A1 (en) | 2011-10-27 |
AU745386B2 (en) | 2002-03-21 |
EP1005376A4 (de) | 2004-11-24 |
US7998734B2 (en) | 2011-08-16 |
ATE486614T1 (de) | 2010-11-15 |
ES2355588T3 (es) | 2011-03-29 |
US20080213222A1 (en) | 2008-09-04 |
JP4289687B2 (ja) | 2009-07-01 |
EP1005376B1 (de) | 2010-11-03 |
EP1005376A1 (de) | 2000-06-07 |
AU6458698A (en) | 1998-10-12 |
US6093392A (en) | 2000-07-25 |
CA2283639A1 (en) | 1998-09-24 |
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