CA2283639A1 - Methods and compositions for use in gene therapy for treatment of hemophilia - Google Patents
Methods and compositions for use in gene therapy for treatment of hemophilia Download PDFInfo
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- CA2283639A1 CA2283639A1 CA002283639A CA2283639A CA2283639A1 CA 2283639 A1 CA2283639 A1 CA 2283639A1 CA 002283639 A CA002283639 A CA 002283639A CA 2283639 A CA2283639 A CA 2283639A CA 2283639 A1 CA2283639 A1 CA 2283639A1
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- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- 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)
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- 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)
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US4071197P | 1997-03-14 | 1997-03-14 | |
| US60/040,711 | 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 |
|---|---|
| CA2283639A1 true CA2283639A1 (en) | 1998-09-24 |
Family
ID=21912503
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002283639A Abandoned CA2283639A1 (en) | 1997-03-14 | 1998-03-12 | Methods and compositions for use in gene therapy for treatment of hemophilia |
Country Status (9)
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|---|---|
| US (3) | US6093392A (https=) |
| EP (1) | EP1005376B1 (https=) |
| JP (1) | JP4289687B2 (https=) |
| AT (1) | ATE486614T1 (https=) |
| AU (1) | AU745386B2 (https=) |
| CA (1) | CA2283639A1 (https=) |
| DE (1) | DE69841982D1 (https=) |
| ES (1) | ES2355588T3 (https=) |
| WO (1) | WO1998041240A1 (https=) |
Families Citing this family (57)
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| US5885971A (en) * | 1995-03-24 | 1999-03-23 | The Regents Of The University Of California | Gene therapy by secretory gland expression |
| US6531455B1 (en) * | 1995-03-24 | 2003-03-11 | The Regents Of The University Of California | Delivery of polynucleotides by secretory gland expression |
| US20020037867A1 (en) * | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | 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 |
| EP1082413B1 (en) * | 1998-05-28 | 2008-07-23 | 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 |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US6503887B1 (en) * | 1999-02-19 | 2003-01-07 | Matthew During | Peroral gene therapy of diabetes and obesity |
| WO2000074688A1 (en) * | 1999-06-08 | 2000-12-14 | The Children's Hospital Of Philadelphia | Methods for preventing formation of inhibitory antibodies in the setting of gene therapy |
| 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 |
| WO2001049118A1 (en) * | 2000-01-03 | 2001-07-12 | 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 |
| US7201898B2 (en) * | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| US20020106381A1 (en) * | 2000-06-13 | 2002-08-08 | High Katherine A. | 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 |
| EP1418810A4 (en) | 2001-08-03 | 2006-08-02 | Us Gov Health & Human Serv | TREATMENT OF HEMOPHILIA ORAL |
| PT1567641E (pt) | 2001-08-24 | 2012-08-21 | Uvic Industry Partnerships Inc | Proaerolisina contendo sequência de activação de proteases e métodos de uso para o tratamento do cancro da próstata |
| 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 |
| WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
| SI3235827T1 (sl) | 2003-06-19 | 2021-05-31 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| 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 |
| WO2005077333A2 (en) * | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| US7722596B2 (en) * | 2004-02-26 | 2010-05-25 | Osprey Medical, Inc. | Regional cardiac tissue treatment |
| EP1804839B1 (en) | 2004-09-22 | 2012-03-14 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| WO2006089340A2 (en) * | 2005-02-23 | 2006-08-31 | V-Kardia Pty Ltd | Polynucleotide delivery to cardiac tissue |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And 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 |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | 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 |
| KR20130028044A (ko) * | 2009-12-08 | 2013-03-18 | 헬스 리서치 인코포레이티드 | 보조제로서 퍼옥시레독신1 (ｐｒｘ１)을 사용하는 방법 및 조성물 |
| US9295816B2 (en) * | 2009-12-09 | 2016-03-29 | Osprey Medical, Inc. | Catheter with distal and proximal ports |
| DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| EP2627665B1 (en) | 2010-10-12 | 2015-12-16 | The Children's Hospital Of Philadelphia | Methods and compositions for treating hemophilia B |
| PL3586861T3 (pl) | 2011-06-08 | 2022-05-23 | Translate Bio, Inc. | Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna |
| WO2012177778A1 (en) | 2011-06-20 | 2012-12-27 | Mount Sinai School Of Medicine | Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders |
| US9211372B2 (en) | 2011-08-11 | 2015-12-15 | Osprey Medical, Inc. | Systems and methods for limb treatment |
| HK1222673A1 (zh) * | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| BR112016019940A2 (pt) | 2014-03-21 | 2017-10-24 | Univ Leland Stanford Junior | edição de genoma sem nucleases |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| WO2017100676A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| KR102415992B1 (ko) | 2016-04-15 | 2022-07-04 | 다케다 야쿠힌 고교 가부시키가이샤 | 약물동역학 약물 투약 요법을 제공하는 방법 및 장치 |
| US10896749B2 (en) | 2017-01-27 | 2021-01-19 | Shire Human Genetic Therapies, Inc. | Drug monitoring tool |
| CN110945127B (zh) | 2017-05-22 | 2024-07-12 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| 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 |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
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| DE3380848D1 (en) * | 1982-08-04 | 1989-12-21 | Nat Res Dev | Molecular cloning of the gene for human anti-haemophilic factor 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 |
| EP0932418B1 (en) * | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| EP0928202A4 (en) * | 1996-09-25 | 2001-09-05 | Cell Genesys Inc | NON-INVASIVE ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS |
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1998
- 1998-03-12 DE DE69841982T patent/DE69841982D1/de not_active Expired - Lifetime
- 1998-03-12 JP JP54061098A patent/JP4289687B2/ja not_active Expired - Lifetime
- 1998-03-12 AT AT98910320T patent/ATE486614T1/de not_active IP Right Cessation
- 1998-03-12 WO PCT/US1998/004790 patent/WO1998041240A1/en not_active Ceased
- 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 CA CA002283639A patent/CA2283639A1/en not_active Abandoned
- 1998-03-12 EP EP98910320A patent/EP1005376B1/en not_active Expired - Lifetime
- 1998-03-12 US US09/038,910 patent/US6093392A/en not_active Expired - Lifetime
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2007
- 2007-10-31 US US11/932,017 patent/US7998734B2/en not_active Expired - Fee Related
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2011
- 2011-07-08 US US13/178,992 patent/US20110263690A1/en not_active Abandoned
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| Publication number | Publication date |
|---|---|
| US20110263690A1 (en) | 2011-10-27 |
| US7998734B2 (en) | 2011-08-16 |
| AU745386B2 (en) | 2002-03-21 |
| EP1005376A4 (en) | 2004-11-24 |
| JP2001517221A (ja) | 2001-10-02 |
| US6093392A (en) | 2000-07-25 |
| EP1005376B1 (en) | 2010-11-03 |
| AU6458698A (en) | 1998-10-12 |
| ES2355588T3 (es) | 2011-03-29 |
| EP1005376A1 (en) | 2000-06-07 |
| US20080213222A1 (en) | 2008-09-04 |
| WO1998041240A1 (en) | 1998-09-24 |
| ATE486614T1 (de) | 2010-11-15 |
| JP4289687B2 (ja) | 2009-07-01 |
| DE69841982D1 (de) | 2010-12-16 |
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