AU745386B2 - 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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- AU745386B2 AU745386B2 AU64586/98A AU6458698A AU745386B2 AU 745386 B2 AU745386 B2 AU 745386B2 AU 64586/98 A AU64586/98 A AU 64586/98A AU 6458698 A AU6458698 A AU 6458698A AU 745386 B2 AU745386 B2 AU 745386B2
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- 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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- 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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- 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
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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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- Pharmacology & Pharmacy (AREA)
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Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU48844/02A AU779514C (en) | 1997-03-14 | 2002-06-19 | Methods and compositions for use in gene therapy for treatment of hemophilia |
| AU2005201735A AU2005201735B2 (en) | 1997-03-14 | 2005-04-26 | Methods and compositions for use in gene therapy for treatment of Hemophilia |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US4071197P | 1997-03-14 | 1997-03-14 | |
| US60/040711 | 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU48844/02A Division AU779514C (en) | 1997-03-14 | 2002-06-19 | Methods and compositions for use in gene therapy for treatment of hemophilia |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU6458698A AU6458698A (en) | 1998-10-12 |
| AU745386B2 true AU745386B2 (en) | 2002-03-21 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| AU64586/98A Expired AU745386B2 (en) | 1997-03-14 | 1998-03-12 | Methods and compositions for use in gene therapy for treatment of hemophilia |
Country Status (9)
| Country | Link |
|---|---|
| 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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| 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 |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, 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 |
| 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 |
| CA2364417C (en) * | 1999-02-19 | 2013-11-05 | Matthew John 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 |
| AU2756301A (en) * | 2000-01-03 | 2001-07-16 | 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 |
| WO2001096587A2 (en) * | 2000-06-13 | 2001-12-20 | The Children's Hospital Of Philadelphia | Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus |
| WO2002040544A2 (en) * | 2000-11-14 | 2002-05-23 | Board Of Regents, University 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 |
| JP4319908B2 (ja) | 2001-08-24 | 2009-08-26 | ユニバーシティ オブ ビクトリア イノベーション アンド ディベロップメント コーポレーション | プロテアーゼ活性化配列を含むプロアエロリシンおよび前立腺癌の治療のための使用方法 |
| 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 |
| SI1633772T1 (sl) | 2003-06-19 | 2016-06-30 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| US8137960B2 (en) | 2003-12-04 | 2012-03-20 | The United States Of America As Represented By The Department Of Health And Human Services | 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 |
| US20060148742A1 (en) * | 2004-02-26 | 2006-07-06 | Kaye David M | Polynucleotide delivery to cardiac tissue |
| WO2006036502A2 (en) | 2004-09-22 | 2006-04-06 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| 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 |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | 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 |
| US9050291B2 (en) * | 2009-12-08 | 2015-06-09 | 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 |
| WO2011094198A1 (en) * | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| US9175280B2 (en) | 2010-10-12 | 2015-11-03 | Sangamo Biosciences, Inc. | Methods and compositions for treating hemophilia B |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| US9155784B2 (en) | 2011-06-20 | 2015-10-13 | Icahn School Of Medicine At Mount Sinai | Anti-TNF-α 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 |
| ES2934848T3 (es) * | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| CA3020346A1 (en) | 2016-04-15 | 2017-10-19 | Baxalta Incorporated | Method and apparatus for providing a pharmacokinetic drug dosing regimen |
| 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 |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
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| EP0107278B1 (en) * | 1982-08-04 | 1989-11-15 | National Research Development Corporation | 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 |
| US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| US5817784A (en) * | 1996-08-09 | 1998-10-06 | Amgen Inc. | Neurogene |
| JP2001500497A (ja) * | 1996-09-06 | 2001-01-16 | 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 | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| 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 |
| 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 EP EP98910320A patent/EP1005376B1/en not_active Expired - Lifetime
- 1998-03-12 JP JP54061098A patent/JP4289687B2/ja not_active Expired - Lifetime
- 1998-03-12 US US09/038,910 patent/US6093392A/en not_active Expired - Lifetime
- 1998-03-12 AU AU64586/98A patent/AU745386B2/en not_active Expired
- 1998-03-12 WO PCT/US1998/004790 patent/WO1998041240A1/en not_active Ceased
- 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 ES ES98910320T patent/ES2355588T3/es not_active Expired - Lifetime
- 1998-03-12 CA CA002283639A patent/CA2283639A1/en not_active Abandoned
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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
Non-Patent Citations (2)
| Title |
|---|
| CHEN L. ET AL (1997) HUM GENE THER 8(2):125-35 * |
| KOEBERL D. D. ET AL (1997) PROC NATL ACAD SCI USA 94:1426-31 * |
Also Published As
| Publication number | Publication date |
|---|---|
| AU6458698A (en) | 1998-10-12 |
| EP1005376A1 (en) | 2000-06-07 |
| JP2001517221A (ja) | 2001-10-02 |
| DE69841982D1 (de) | 2010-12-16 |
| US20110263690A1 (en) | 2011-10-27 |
| CA2283639A1 (en) | 1998-09-24 |
| US20080213222A1 (en) | 2008-09-04 |
| ES2355588T3 (es) | 2011-03-29 |
| EP1005376B1 (en) | 2010-11-03 |
| JP4289687B2 (ja) | 2009-07-01 |
| US7998734B2 (en) | 2011-08-16 |
| WO1998041240A1 (en) | 1998-09-24 |
| EP1005376A4 (en) | 2004-11-24 |
| US6093392A (en) | 2000-07-25 |
| ATE486614T1 (de) | 2010-11-15 |
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