DE69930625D1 - Rekombinante aav-vektoren für die gentherapie von hämophilie a - Google Patents
Rekombinante aav-vektoren für die gentherapie von hämophilie aInfo
- Publication number
- DE69930625D1 DE69930625D1 DE69930625T DE69930625T DE69930625D1 DE 69930625 D1 DE69930625 D1 DE 69930625D1 DE 69930625 T DE69930625 T DE 69930625T DE 69930625 T DE69930625 T DE 69930625T DE 69930625 D1 DE69930625 D1 DE 69930625D1
- Authority
- DE
- Germany
- Prior art keywords
- hemophilia
- gene therapy
- factor viii
- aav vectors
- recombinant aav
- 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 - Fee Related
Links
- 208000031220 Hemophilia Diseases 0.000 title 1
- 208000009292 Hemophilia A Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 102000001690 Factor VIII Human genes 0.000 abstract 3
- 108010054218 Factor VIII Proteins 0.000 abstract 3
- 229960000301 factor viii Drugs 0.000 abstract 3
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 1
- 102000057593 human F8 Human genes 0.000 abstract 1
- 229960000900 human factor viii Drugs 0.000 abstract 1
- 239000000463 material Substances 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
Classifications
-
- 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
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US84425 | 1998-05-27 | ||
| US09/084,425 US6797505B2 (en) | 1998-05-27 | 1998-05-27 | Recombinant AAV vectors for gene therapy of hemophilia A |
| PCT/US1999/010473 WO1999061642A1 (en) | 1998-05-27 | 1999-05-27 | Recombinant aav vectors for gene therapy of hemophilia a |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DE69930625D1 true DE69930625D1 (de) | 2006-05-18 |
| DE69930625T2 DE69930625T2 (de) | 2007-01-25 |
Family
ID=22184892
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DE69930625T Expired - Fee Related DE69930625T2 (de) | 1998-05-27 | 1999-05-27 | Rekombinante aav-vektoren für die gentherapie von hämophilie a |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US6797505B2 (de) |
| EP (1) | EP1082446B1 (de) |
| JP (1) | JP2002516114A (de) |
| AT (1) | ATE321873T1 (de) |
| AU (1) | AU775423B2 (de) |
| CA (1) | CA2329505A1 (de) |
| DE (1) | DE69930625T2 (de) |
| WO (1) | WO1999061642A1 (de) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| DE69941905D1 (de) * | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US6759050B1 (en) * | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| WO2001027303A1 (en) * | 1999-10-12 | 2001-04-19 | The University Of North Carolina At Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| EP1240345A2 (de) * | 1999-12-10 | 2002-09-18 | Ariad Gene Therapeutics, Inc. | Verfahren zur genexpression in primaten |
| JP4085231B2 (ja) | 2000-02-28 | 2008-05-14 | 株式会社ビークル | タンパク質中空ナノ粒子とそれを用いた物質運搬体、ならびに細胞への物質導入方法 |
| WO2002006214A1 (en) * | 2000-07-19 | 2002-01-24 | Mitsubishi Pharma Corporation | Sulfonic acid derivatives of hydroxamic acids and their use as medicinal products |
| KR101004086B1 (ko) | 2002-06-18 | 2010-12-27 | 에자이 알앤드디 매니지먼트 가부시키가이샤 | 유전자 치료용 초대 배양 지방세포 |
| CN1498964A (zh) * | 2002-11-07 | 2004-05-26 | 本元正阳基因技术股份有限公司 | 可诱导RNAi途径的用于基因治疗的系列重组腺相关病毒 |
| US20040092008A1 (en) * | 2002-11-12 | 2004-05-13 | Snyder Richard O. | Recombinant AAV vectors for gene therapy of hemophilia A |
| US7485291B2 (en) | 2003-06-03 | 2009-02-03 | Cell Genesys, Inc. | Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof |
| US7709224B2 (en) | 2003-06-03 | 2010-05-04 | Biosante Pharmaceuticals, Inc. | Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| KR102450833B1 (ko) | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| US20250352626A1 (en) * | 2022-06-10 | 2025-11-20 | University Of Florida Research Foundation, Incorporated | Immune tolerance induction to viral capsids |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
| 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 |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| 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 |
| EP1005376B1 (de) * | 1997-03-14 | 2010-11-03 | The Children's Hospital of Philadelphia | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
| US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
-
1998
- 1998-05-27 US US09/084,425 patent/US6797505B2/en not_active Expired - Fee Related
-
1999
- 1999-05-27 AT AT99925607T patent/ATE321873T1/de not_active IP Right Cessation
- 1999-05-27 CA CA002329505A patent/CA2329505A1/en not_active Abandoned
- 1999-05-27 WO PCT/US1999/010473 patent/WO1999061642A1/en not_active Ceased
- 1999-05-27 DE DE69930625T patent/DE69930625T2/de not_active Expired - Fee Related
- 1999-05-27 AU AU41857/99A patent/AU775423B2/en not_active Ceased
- 1999-05-27 JP JP2000551025A patent/JP2002516114A/ja active Pending
- 1999-05-27 EP EP99925607A patent/EP1082446B1/de not_active Expired - Lifetime
Also Published As
| Publication number | Publication date |
|---|---|
| US6797505B2 (en) | 2004-09-28 |
| EP1082446A1 (de) | 2001-03-14 |
| WO1999061642A1 (en) | 1999-12-02 |
| AU775423B2 (en) | 2004-07-29 |
| JP2002516114A (ja) | 2002-06-04 |
| ATE321873T1 (de) | 2006-04-15 |
| AU4185799A (en) | 1999-12-13 |
| CA2329505A1 (en) | 1999-12-02 |
| EP1082446B1 (de) | 2006-03-29 |
| DE69930625T2 (de) | 2007-01-25 |
| US20020155580A1 (en) | 2002-10-24 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| 8364 | No opposition during term of opposition | ||
| 8339 | Ceased/non-payment of the annual fee |