JP2008540445A5 - - Google Patents
Download PDFInfo
- 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
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 Expired - Lifetime 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 Expired - Lifetime 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 Expired - Lifetime 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) | EP1879624B1 (enExample) |
| JP (3) | JP5829372B2 (enExample) |
| CN (4) | CN104306986A (enExample) |
| AT (1) | ATE525092T1 (enExample) |
| AU (1) | AU2006243776A1 (enExample) |
| BR (1) | BRPI0611379A2 (enExample) |
| CA (2) | CA2607173C (enExample) |
| ES (1) | ES2887076T3 (enExample) |
| IL (2) | IL187078A (enExample) |
| PL (4) | PL2420256T3 (enExample) |
| PT (4) | PT3520823T (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 |
| AU2006242129A1 (en) * | 2005-05-02 | 2006-11-09 | Genzyme Corporation | Gene therapy for spinal cord disorders |
| PT3520823T (pt) | 2005-05-02 | 2021-09-16 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
| CN101460621B (zh) | 2006-06-07 | 2016-01-27 | 建新公司 | 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗 |
| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| 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 |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| 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 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | 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 |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | 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 |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| CN116019934A (zh) | 2015-02-10 | 2023-04-28 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
| JP6659050B2 (ja) * | 2015-03-26 | 2020-03-04 | スーヂョウ アウゾン バイオロジカル テクノロジー カンパニー， リミテッド | Ｐ７５ｅｃｄ及び／又はｐ７５による神経学的障害の診断又は処置方法 |
| 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 |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| 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 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| ES3065816T3 (en) | 2017-06-07 | 2026-05-08 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| 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) |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| CN113383010B (zh) | 2018-09-28 | 2025-09-09 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
| 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 |
| ATE304604T1 (de) | 1993-06-24 | 2005-09-15 | Frank L Graham | Adenovirus vektoren für gentherapie |
| DE69434594T2 (de) | 1993-10-25 | 2006-09-21 | Canji, Inc., San Diego | Rekombinante adenoviren-vektor und verfahren zur verwendung |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| 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 |
| WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | 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 |
| JP2004516016A (ja) | 2000-09-18 | 2004-06-03 | ジェンザイム・コーポレイション | ハイブリッドユビキチンプロモーターを含む発現ベクター |
| 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 |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| AU2002364221B2 (en) | 2001-12-21 | 2008-07-10 | The Salk Institute For Biological Studies | 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 |
| WO2003100031A2 (en) * | 2002-05-20 | 2003-12-04 | Board Of Regents, The University Of Texas System | Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues |
| ES2562490T3 (es) * | 2003-05-01 | 2016-03-04 | Genzyme Corporation | Terapia génica para trastornos neurometabólicos |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | 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 |
| PT3520823T (pt) | 2005-05-02 | 2021-09-16 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
| AU2006242129A1 (en) | 2005-05-02 | 2006-11-09 | Genzyme Corporation | Gene therapy for spinal cord disorders |
| ES2700048T3 (es) | 2006-02-08 | 2019-02-13 | Genzyme Corp | Terapia génica par la enfermedad de Niemann-Pick tipo A |
| US20150030561A1 (en) | 2011-05-16 | 2015-01-29 | Genzyme Corp. | Use of CXCR4 Antagonists |
| 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 PT PT19153520T patent/PT3520823T/pt unknown
- 2006-05-02 WO PCT/US2006/017242 patent/WO2006119458A1/en not_active Ceased
- 2006-05-02 PL PL11169633.2T patent/PL2420256T3/pl unknown
- 2006-05-02 CA CA2607173A patent/CA2607173C/en not_active Expired - Lifetime
- 2006-05-02 JP JP2008510224A patent/JP5829372B2/ja not_active Expired - Lifetime
- 2006-05-02 AU AU2006243776A patent/AU2006243776A1/en not_active Abandoned
- 2006-05-02 ES ES19153520T patent/ES2887076T3/es not_active Expired - Lifetime
- 2006-05-02 BR BRPI0611379-6A patent/BRPI0611379A2/pt not_active Application Discontinuation
- 2006-05-02 EP EP06759081A patent/EP1879624B1/en not_active Expired - Lifetime
- 2006-05-02 PT PT06759081T patent/PT1879624E/pt unknown
- 2006-05-02 CN CN201410483749.6A patent/CN104306986A/zh active Pending
- 2006-05-02 EP EP16162716.1A patent/EP3058959B1/en not_active Expired - Lifetime
- 2006-05-02 CN CNA2006800237754A patent/CN101212988A/zh active Pending
- 2006-05-02 CN CN201611031204.7A patent/CN107007842A/zh active Pending
- 2006-05-02 PL PL19153520T patent/PL3520823T3/pl unknown
- 2006-05-02 EP EP19153520.2A patent/EP3520823B1/en not_active Expired - Lifetime
- 2006-05-02 PL PL16162716T patent/PL3058959T3/pl unknown
- 2006-05-02 AT AT06759081T patent/ATE525092T1/de not_active IP Right Cessation
- 2006-05-02 PL PL06759081T patent/PL1879624T3/pl unknown
- 2006-05-02 PT PT111696332T patent/PT2420256T/pt unknown
- 2006-05-02 CA CA2998603A patent/CA2998603C/en not_active Expired - Lifetime
- 2006-05-02 CN CN201710506914.9A patent/CN107362371A/zh active Pending
- 2006-05-02 EP EP11169633.2A patent/EP2420256B1/en not_active Expired - Lifetime
- 2006-05-02 PT PT16162716T patent/PT3058959T/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 not_active Expired - Lifetime
-
2020
- 2020-03-03 US US16/808,206 patent/US11957765B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11957765B2 (en) | Gene therapy for neurometabolic disorders | |
| JP2009526067A5 (enExample) | ||
| Zhang et al. | Several rAAV vectors efficiently cross the blood–brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system | |
| Cheng et al. | Gene therapy progress and prospects: gene therapy of lysosomal storage disorders | |
| JP6312771B2 (ja) | 神経代謝性疾患についての遺伝子治療 | |
| EP3498851B1 (en) | Cns gene delivery using peripheral administration of aav vectors | |
| TWI547288B (zh) | 用於治療疾病之載體及序列 | |
| Miyake et al. | Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors | |
| JP2016503405A5 (enExample) | ||
| EA036394B1 (ru) | Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний | |
| Gomez Limia et al. | Emerging perspectives on gene therapy delivery for neurodegenerative and neuromuscular disorders | |
| WO2005120581A2 (en) | Gene therapy for neurometabolic disorders | |
| Hironaka et al. | Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector | |
| Ruzo et al. | Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice | |
| Hildinger et al. | Advances in AAV-mediated gene transfer for the treatment of inherited disorders | |
| Cardone | Prospects for gene therapy in inherited neurodegenerative diseases | |
| US20230277687A1 (en) | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy | |
| Weismann | Approaches and Considerations Towards a Safe and Effective Adeno-Associated Virus Mediated Therapeutic Intervention for GM1-Gangliosidosis: A Dissertation | |
| Nandi et al. | Revolutionizing Gene Therapy: An Explicative Review on Engineering Adeno-associated Virus Vectors | |
| McCarty et al. | Gene Therapy for Neurodevelopmental Disorders | |
| Murlidharan et al. | Gene Therapy of CNS Disorders Using Recombinant AAV Vectors | |
| Cearley | Adeno-associated virus vector mediated correction of a neurogenetic disease: Influence of the vector capsid on dispersal of a therapeutic gene within the central nervous system | |
| Broekman | Discussion and conclusion | |
| Birmingham et al. | 507. Modeling Adeno-Associated Virus 2 Capsid for the Identification of Targeting Ligands for Enhanced Gene Transfer to Polarized Human Airway Epithelial Cells |