JP2008540445A5 - - Google Patents
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- JP2008540445A5 JP2008540445A5 JP2008510224A JP2008510224A JP2008540445A5 JP 2008540445 A5 JP2008540445 A5 JP 2008540445A5 JP 2008510224 A JP2008510224 A JP 2008510224A JP 2008510224 A JP2008510224 A JP 2008510224A JP 2008540445 A5 JP2008540445 A5 JP 2008540445A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- site
- aav vector
- aav2
- 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.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims 37
- 239000013607 AAV vector Substances 0.000 claims 17
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 10
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 6
- 210000003169 central nervous system Anatomy 0.000 claims 6
- 108090000604 Hydrolases Proteins 0.000 claims 4
- 102000004157 Hydrolases Human genes 0.000 claims 4
- 241000124008 Mammalia Species 0.000 claims 4
- 230000002132 lysosomal effect Effects 0.000 claims 4
- 102000005593 Endopeptidases Human genes 0.000 claims 3
- 108010059378 Endopeptidases Proteins 0.000 claims 3
- 210000004556 brain Anatomy 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 210000001638 cerebellum Anatomy 0.000 claims 3
- 239000002184 metal Substances 0.000 claims 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 2
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 210000003591 cerebellar nuclei Anatomy 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 210000001320 hippocampus Anatomy 0.000 claims 2
- 210000001577 neostriatum Anatomy 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 210000000278 spinal cord Anatomy 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000035343 Infantile neurovisceral acid sphingomyelinase deficiency Diseases 0.000 claims 1
- 102000003729 Neprilysin Human genes 0.000 claims 1
- 108090000028 Neprilysin Proteins 0.000 claims 1
- 201000000794 Niemann-Pick disease type A Diseases 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 208000007824 Type A Niemann-Pick Disease Diseases 0.000 claims 1
- 230000002159 abnormal effect Effects 0.000 claims 1
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 1
- 210000000133 brain stem Anatomy 0.000 claims 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- 210000001947 dentate gyrus Anatomy 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 210000001353 entorhinal cortex Anatomy 0.000 claims 1
- 210000001652 frontal lobe Anatomy 0.000 claims 1
- 210000003016 hypothalamus Anatomy 0.000 claims 1
- 210000001259 mesencephalon Anatomy 0.000 claims 1
- 208000030159 metabolic disease Diseases 0.000 claims 1
- 230000004060 metabolic process Effects 0.000 claims 1
- 210000000869 occipital lobe Anatomy 0.000 claims 1
- 108010032563 oligopeptidase Proteins 0.000 claims 1
- 210000001152 parietal lobe Anatomy 0.000 claims 1
- BULVZWIRKLYCBC-UHFFFAOYSA-N phorate Chemical compound CCOP(=S)(OCC)SCSCC BULVZWIRKLYCBC-UHFFFAOYSA-N 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 210000002975 pon Anatomy 0.000 claims 1
- 210000003523 substantia nigra Anatomy 0.000 claims 1
- 210000003478 temporal lobe Anatomy 0.000 claims 1
- 210000001103 thalamus Anatomy 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US67705705P | 2005-05-02 | 2005-05-02 | |
| US60/677,057 | 2005-05-02 | ||
| US68580805P | 2005-05-31 | 2005-05-31 | |
| US60/685,808 | 2005-05-31 | ||
| PCT/US2006/017242 WO2006119458A1 (en) | 2005-05-02 | 2006-05-02 | Gene therapy for neurometabolic disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013201138A Division JP2014037418A (ja) | 2005-05-02 | 2013-09-27 | 神経代謝性疾患のための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2008540445A JP2008540445A (ja) | 2008-11-20 |
| JP2008540445A5 true JP2008540445A5 (enExample) | 2009-07-09 |
| JP5829372B2 JP5829372B2 (ja) | 2015-12-09 |
Family
ID=37308324
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008510224A Active JP5829372B2 (ja) | 2005-05-02 | 2006-05-02 | 神経代謝性疾患のための遺伝子治療 |
| JP2013201138A Pending JP2014037418A (ja) | 2005-05-02 | 2013-09-27 | 神経代謝性疾患のための遺伝子治療 |
| JP2015197051A Active JP6338560B2 (ja) | 2005-05-02 | 2015-10-02 | 神経代謝性疾患のための遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013201138A Pending JP2014037418A (ja) | 2005-05-02 | 2013-09-27 | 神経代謝性疾患のための遺伝子治療 |
| JP2015197051A Active JP6338560B2 (ja) | 2005-05-02 | 2015-10-02 | 神経代謝性疾患のための遺伝子治療 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US10632213B2 (enExample) |
| EP (4) | EP3058959B1 (enExample) |
| JP (3) | JP5829372B2 (enExample) |
| CN (4) | CN107007842A (enExample) |
| AT (1) | ATE525092T1 (enExample) |
| AU (1) | AU2006243776A1 (enExample) |
| BR (1) | BRPI0611379A2 (enExample) |
| CA (2) | CA2998603C (enExample) |
| ES (1) | ES2887076T3 (enExample) |
| IL (2) | IL187078A (enExample) |
| PL (4) | PL2420256T3 (enExample) |
| PT (4) | PT3058959T (enExample) |
| WO (1) | WO2006119458A1 (enExample) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3302529B2 (ja) | 1995-04-28 | 2002-07-15 | 株式会社クボタ | ポンプ吸水槽の整流装置 |
| MX2007013734A (es) * | 2005-05-02 | 2008-03-14 | Genzyme Corp | Terapia genica para trastornos de la medula espinal. |
| CN107007842A (zh) | 2005-05-02 | 2017-08-04 | 建新公司 | 神经代谢疾病的基因治疗 |
| ES2596885T3 (es) | 2006-06-07 | 2017-01-12 | Genzyme Corporation | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal |
| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| EP2352847B1 (en) | 2008-11-10 | 2014-01-08 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Gene signature for predicting prognosis of patients with solid tumors |
| RS67077B1 (sr) | 2009-05-02 | 2025-08-29 | Genzyme Corp | Genska terapija za neurodegenerativne poremećaje |
| US9150926B2 (en) | 2010-12-06 | 2015-10-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Diagnosis and treatment of adrenocortical tumors using human microRNA-483 |
| WO2012145646A1 (en) * | 2011-04-20 | 2012-10-26 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016081924A1 (en) * | 2014-11-20 | 2016-05-26 | Duke University | Compositions, systems and methods for cell therapy |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| AU2016219398A1 (en) | 2015-02-10 | 2017-09-28 | Genzyme Corporation | Enhanced delivery of viral particles to the striatum and cortex |
| US11046746B2 (en) | 2015-03-26 | 2021-06-29 | Suzhou Auzone Biological Technology Co., Ltd | Method of treatment of cerebral amyloid angiopathy with P75ECD peptide and/or P75ECD-FC fusion protein |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| WO2016196507A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| CN110809583A (zh) | 2017-06-07 | 2020-02-18 | 瑞泽恩制药公司 | 用于内化酶的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| KR20200118151A (ko) | 2018-02-07 | 2020-10-14 | 리제너론 파마슈티칼스 인코포레이티드 | 치료 단백질 전달을 위한 방법 및 조성물 |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| MX2020012350A (es) | 2018-05-17 | 2021-01-29 | Regeneron Pharma | Anticuerpos anti-cd63, conjugados y usos de estos. |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| KR20240126916A (ko) * | 2023-02-14 | 2024-08-22 | 경북대학교 산학협력단 | Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE8303626D0 (sv) | 1983-06-23 | 1983-06-23 | Kabigen Ab | A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced |
| US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| JP3532566B2 (ja) | 1993-06-24 | 2004-05-31 | エル． グラハム，フランク | 遺伝子治療のためのアデノウイルスベクター |
| JP3875990B2 (ja) | 1993-10-25 | 2007-01-31 | カンジ，インコーポレイテッド | 組換えアデノウイルスベクターおよび使用方法 |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| EP0755454B1 (en) | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
| WO1997039789A1 (en) | 1996-04-22 | 1997-10-30 | Medtronic, Inc. | Two-stage angled venous cannula |
| US6544785B1 (en) | 1998-09-14 | 2003-04-08 | Mount Sinai School Of Medicine Of New York University | Helper-free rescue of recombinant negative strand RNA viruses |
| US6582692B1 (en) * | 1999-11-17 | 2003-06-24 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| US20030165481A1 (en) * | 2000-02-24 | 2003-09-04 | Hersh Louis B. | Amyloid peptide inactivating enzyme to treat Alzheimer's disease |
| EP1319082B1 (en) | 2000-09-18 | 2005-11-16 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| US20030050273A1 (en) * | 2001-08-29 | 2003-03-13 | Keiya Ozawa | Compositions and methods for treating neurodegenerative diseases |
| 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 |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| NZ533833A (en) | 2001-12-21 | 2008-01-31 | Salk Inst For Biological Studi | Targeted retrograde gene delivery to motor neurons |
| US6998118B2 (en) | 2001-12-21 | 2006-02-14 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery for neuronal protection |
| AU2003220115A1 (en) * | 2002-05-20 | 2003-12-12 | Board Of Regents, The University Of Texas System | Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| PL1620133T3 (pl) * | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
| US7740168B2 (en) | 2003-08-18 | 2010-06-22 | Visa U.S.A. Inc. | Method and system for generating a dynamic verification value |
| AU2003272868A1 (en) | 2003-10-15 | 2005-04-27 | Agtc Gene Technology Company Ltd. | Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors |
| EP1716870A1 (en) | 2005-04-29 | 2006-11-02 | Bracco Imaging S.p.A. | MRI contrast agents endowed with concentration independent responsiveness |
| CN107007842A (zh) | 2005-05-02 | 2017-08-04 | 建新公司 | 神经代谢疾病的基因治疗 |
| MX2007013734A (es) | 2005-05-02 | 2008-03-14 | Genzyme Corp | Terapia genica para trastornos de la medula espinal. |
| AR059371A1 (es) | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
| EP2709991B1 (en) | 2011-05-16 | 2020-09-02 | Genzyme Corporation | Use of cxcr4 antagonists for treating WHIM syndrome, myelokathexis, neutropenia and lymphocytopenia |
| TWI821227B (zh) | 2017-12-22 | 2023-11-11 | 美商健臻公司 | 胺甲喋呤誘發的免疫耐受性之生物標記 |
-
2006
- 2006-05-02 CN CN201611031204.7A patent/CN107007842A/zh active Pending
- 2006-05-02 PL PL11169633.2T patent/PL2420256T3/pl unknown
- 2006-05-02 ES ES19153520T patent/ES2887076T3/es active Active
- 2006-05-02 EP EP16162716.1A patent/EP3058959B1/en active Active
- 2006-05-02 CN CN201410483749.6A patent/CN104306986A/zh active Pending
- 2006-05-02 BR BRPI0611379-6A patent/BRPI0611379A2/pt not_active Application Discontinuation
- 2006-05-02 AT AT06759081T patent/ATE525092T1/de not_active IP Right Cessation
- 2006-05-02 PT PT16162716T patent/PT3058959T/pt unknown
- 2006-05-02 PT PT19153520T patent/PT3520823T/pt unknown
- 2006-05-02 PT PT06759081T patent/PT1879624E/pt unknown
- 2006-05-02 PL PL16162716T patent/PL3058959T3/pl unknown
- 2006-05-02 EP EP06759081A patent/EP1879624B1/en active Active
- 2006-05-02 EP EP19153520.2A patent/EP3520823B1/en active Active
- 2006-05-02 PT PT111696332T patent/PT2420256T/pt unknown
- 2006-05-02 AU AU2006243776A patent/AU2006243776A1/en not_active Abandoned
- 2006-05-02 CA CA2998603A patent/CA2998603C/en active Active
- 2006-05-02 WO PCT/US2006/017242 patent/WO2006119458A1/en not_active Ceased
- 2006-05-02 CN CNA2006800237754A patent/CN101212988A/zh active Pending
- 2006-05-02 PL PL06759081T patent/PL1879624T3/pl unknown
- 2006-05-02 PL PL19153520T patent/PL3520823T3/pl unknown
- 2006-05-02 CA CA2607173A patent/CA2607173C/en active Active
- 2006-05-02 JP JP2008510224A patent/JP5829372B2/ja active Active
- 2006-05-02 CN CN201710506914.9A patent/CN107362371A/zh active Pending
- 2006-05-02 EP EP11169633.2A patent/EP2420256B1/en active Active
-
2007
- 2007-11-01 IL IL187078A patent/IL187078A/en not_active IP Right Cessation
- 2007-11-02 US US11/934,325 patent/US10632213B2/en active Active
-
2013
- 2013-09-27 JP JP2013201138A patent/JP2014037418A/ja active Pending
-
2015
- 2015-06-03 IL IL239167A patent/IL239167A0/en unknown
- 2015-10-02 JP JP2015197051A patent/JP6338560B2/ja active Active
-
2020
- 2020-03-03 US US16/808,206 patent/US11957765B2/en active Active
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