JP2008540445A5 - - Google Patents
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- Publication number
- 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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (4) | EP3058959B1 (cg-RX-API-DMAC7.html) |
| JP (3) | JP5829372B2 (cg-RX-API-DMAC7.html) |
| CN (4) | CN104306986A (cg-RX-API-DMAC7.html) |
| AT (1) | ATE525092T1 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2006243776A1 (cg-RX-API-DMAC7.html) |
| BR (1) | BRPI0611379A2 (cg-RX-API-DMAC7.html) |
| CA (2) | CA2607173C (cg-RX-API-DMAC7.html) |
| ES (1) | ES2887076T3 (cg-RX-API-DMAC7.html) |
| IL (2) | IL187078A (cg-RX-API-DMAC7.html) |
| PL (4) | PL1879624T3 (cg-RX-API-DMAC7.html) |
| PT (4) | PT2420256T (cg-RX-API-DMAC7.html) |
| WO (1) | WO2006119458A1 (cg-RX-API-DMAC7.html) |
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 |
| JP5766389B2 (ja) * | 2005-05-02 | 2015-08-19 | ジェンザイム・コーポレーション | 脊髄疾患の遺伝子療法 |
| AU2006243776A1 (en) | 2005-05-02 | 2006-11-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| PL2489733T3 (pl) | 2006-06-07 | 2019-08-30 | Genzyme Corporation | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
| SI2158322T1 (sl) * | 2007-06-06 | 2017-10-30 | Genzyme Corporation | Genska terapija za bolezni lizosomskega shranjevanja |
| EP2687609B1 (en) | 2008-11-10 | 2017-01-04 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Method for treating solid tumor |
| BRPI1010868A2 (pt) | 2009-05-02 | 2018-06-12 | Genzyme Corp | terapia gênica para distúrbios neurodegenerativos |
| 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 |
| US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| DK3218386T3 (da) | 2014-11-14 | 2021-06-07 | Voyager Therapeutics Inc | Modulatorisk polynukleotid |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11319555B2 (en) | 2014-11-20 | 2022-05-03 | 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 |
| PL3256594T3 (pl) | 2015-02-10 | 2022-02-14 | Genzyme Corporation | Ulepszone dostarczanie cząstek wirusa do prążkowia i kory |
| CN106794222B (zh) * | 2015-03-26 | 2021-08-24 | 苏州澳宗生物科技有限公司 | 使用p75ecd和/或p75诊断或治疗神经障碍的方法 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| US11027024B2 (en) | 2015-05-29 | 2021-06-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11951121B2 (en) | 2016-05-18 | 2024-04-09 | Voyager Therapeutics, Inc. | Compositions and methods for treating Huntington's disease |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN119491003A (zh) | 2017-05-05 | 2025-02-21 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP7348844B2 (ja) | 2017-06-07 | 2023-09-21 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 内部移行酵素のための組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| TWI722310B (zh) | 2017-08-03 | 2021-03-21 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018352236B2 (en) | 2017-10-16 | 2025-04-10 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
| SG11202007363TA (en) | 2018-02-07 | 2020-08-28 | Regeneron Pharma | Methods and compositions for therapeutic protein delivery |
| US12319929B2 (en) | 2018-05-15 | 2025-06-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| US12252544B2 (en) | 2018-05-17 | 2025-03-18 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | 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 |
| DE69434486T2 (de) | 1993-06-24 | 2006-07-06 | Advec Inc. | Adenovirus vektoren für gentherapie |
| BR9407956A (pt) | 1993-10-25 | 1996-11-26 | Canji Inc | Composiçao farmacêutica vetor de express o de adenovirus recombinante e kit par reduzir a proliferaçao de células tumorais |
| ES2216005T3 (es) | 1993-11-09 | 2004-10-16 | Targeted Genetics Corporation | Produccion de titulos elevados de vectores de aav recombinantes. |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| AU1525797A (en) | 1996-04-22 | 1997-11-12 | 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 |
| AU2001290984A1 (en) | 2000-09-18 | 2002-04-02 | 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 |
| EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
| US6998118B2 (en) | 2001-12-21 | 2006-02-14 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery for neuronal protection |
| EP1478236B1 (en) | 2001-12-21 | 2009-02-11 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery to motor neurons |
| 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 |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| US7740168B2 (en) | 2003-08-18 | 2010-06-22 | Visa U.S.A. Inc. | Method and system for generating a dynamic verification value |
| WO2005035743A1 (en) * | 2003-10-15 | 2005-04-21 | Vector 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 |
| JP5766389B2 (ja) | 2005-05-02 | 2015-08-19 | ジェンザイム・コーポレーション | 脊髄疾患の遺伝子療法 |
| AU2006243776A1 (en) | 2005-05-02 | 2006-11-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| WO2007092563A2 (en) | 2006-02-08 | 2007-08-16 | Genzyme Corporation | Gene therapy for niemann-pick disease type a |
| JP2014513727A (ja) | 2011-05-16 | 2014-06-05 | ジェンザイム・コーポレーション | Ｃｘｃｒ４拮抗薬の使用 |
| 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 AU AU2006243776A patent/AU2006243776A1/en not_active Abandoned
- 2006-05-02 PL PL06759081T patent/PL1879624T3/pl unknown
- 2006-05-02 PL PL11169633.2T patent/PL2420256T3/pl unknown
- 2006-05-02 BR BRPI0611379-6A patent/BRPI0611379A2/pt not_active Application Discontinuation
- 2006-05-02 EP EP16162716.1A patent/EP3058959B1/en active Active
- 2006-05-02 ES ES19153520T patent/ES2887076T3/es active Active
- 2006-05-02 PT PT111696332T patent/PT2420256T/pt unknown
- 2006-05-02 CN CN201410483749.6A patent/CN104306986A/zh active Pending
- 2006-05-02 EP EP19153520.2A patent/EP3520823B1/en active Active
- 2006-05-02 PT PT16162716T patent/PT3058959T/pt unknown
- 2006-05-02 CN CN201710506914.9A patent/CN107362371A/zh active Pending
- 2006-05-02 PL PL19153520T patent/PL3520823T3/pl unknown
- 2006-05-02 CA CA2607173A patent/CA2607173C/en active Active
- 2006-05-02 EP EP11169633.2A patent/EP2420256B1/en active Active
- 2006-05-02 PL PL16162716T patent/PL3058959T3/pl unknown
- 2006-05-02 EP EP06759081A patent/EP1879624B1/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 PT PT19153520T patent/PT3520823T/pt unknown
- 2006-05-02 JP JP2008510224A patent/JP5829372B2/ja active Active
- 2006-05-02 AT AT06759081T patent/ATE525092T1/de not_active IP Right Cessation
- 2006-05-02 CN CN201611031204.7A patent/CN107007842A/zh active Pending
- 2006-05-02 CA CA2998603A patent/CA2998603C/en active Active
- 2006-05-02 PT PT06759081T patent/PT1879624E/pt unknown
-
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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