JP2014040433A5 - - Google Patents
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- JP2014040433A5 JP2014040433A5 JP2013196398A JP2013196398A JP2014040433A5 JP 2014040433 A5 JP2014040433 A5 JP 2014040433A5 JP 2013196398 A JP2013196398 A JP 2013196398A JP 2013196398 A JP2013196398 A JP 2013196398A JP 2014040433 A5 JP2014040433 A5 JP 2014040433A5
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- JP
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Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US46719503P | 2003-05-01 | 2003-05-01 | |
| US60/467,195 | 2003-05-01 | ||
| US47572603P | 2003-06-04 | 2003-06-04 | |
| US60/475,726 | 2003-06-04 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011168292A Division JP2011251992A (ja) | 2003-05-01 | 2011-08-01 | 神経代謝性疾患についての遺伝子治療 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015106021A Division JP6170519B2 (ja) | 2003-05-01 | 2015-05-26 | 神経代謝性疾患についての遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014040433A JP2014040433A (ja) | 2014-03-06 |
| JP2014040433A5 true JP2014040433A5 (https=) | 2014-06-26 |
| JP5841574B2 JP5841574B2 (ja) | 2016-01-13 |
Family
ID=33436721
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006514239A Expired - Fee Related JP4979067B2 (ja) | 2003-05-01 | 2004-04-30 | 神経代謝性疾患についての遺伝子治療 |
| JP2011168292A Pending JP2011251992A (ja) | 2003-05-01 | 2011-08-01 | 神経代謝性疾患についての遺伝子治療 |
| JP2013196398A Expired - Fee Related JP5841574B2 (ja) | 2003-05-01 | 2013-09-24 | 神経代謝性疾患についての遺伝子治療 |
| JP2015106021A Expired - Fee Related JP6170519B2 (ja) | 2003-05-01 | 2015-05-26 | 神経代謝性疾患についての遺伝子治療 |
| JP2016205651A Expired - Lifetime JP6312771B2 (ja) | 2003-05-01 | 2016-10-20 | 神経代謝性疾患についての遺伝子治療 |
| JP2018000518A Pending JP2018058901A (ja) | 2003-05-01 | 2018-01-05 | 神経代謝性疾患についての遺伝子治療 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006514239A Expired - Fee Related JP4979067B2 (ja) | 2003-05-01 | 2004-04-30 | 神経代謝性疾患についての遺伝子治療 |
| JP2011168292A Pending JP2011251992A (ja) | 2003-05-01 | 2011-08-01 | 神経代謝性疾患についての遺伝子治療 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015106021A Expired - Fee Related JP6170519B2 (ja) | 2003-05-01 | 2015-05-26 | 神経代謝性疾患についての遺伝子治療 |
| JP2016205651A Expired - Lifetime JP6312771B2 (ja) | 2003-05-01 | 2016-10-20 | 神経代謝性疾患についての遺伝子治療 |
| JP2018000518A Pending JP2018058901A (ja) | 2003-05-01 | 2018-01-05 | 神経代謝性疾患についての遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| EP (3) | EP1620133B1 (https=) |
| JP (6) | JP4979067B2 (https=) |
| CY (1) | CY1117457T1 (https=) |
| DK (1) | DK1620133T3 (https=) |
| ES (1) | ES2562490T3 (https=) |
| HU (1) | HUE026861T2 (https=) |
| IL (2) | IL171635A (https=) |
| PL (1) | PL1620133T3 (https=) |
| PT (1) | PT1620133E (https=) |
| SI (1) | SI1620133T1 (https=) |
| WO (1) | WO2004098648A1 (https=) |
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| US7605249B2 (en) | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| US7618948B2 (en) | 2002-11-26 | 2009-11-17 | Medtronic, Inc. | Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA |
| US7994149B2 (en) | 2003-02-03 | 2011-08-09 | Medtronic, Inc. | Method for treatment of Huntington's disease through intracranial delivery of sirna |
| US7732591B2 (en) | 2003-11-25 | 2010-06-08 | Medtronic, Inc. | Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna |
| 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 |
| CA2606490C (en) | 2005-05-02 | 2018-02-06 | Genzyme Corporation | Gene therapy for spinal cord disorders |
| JP5829372B2 (ja) * | 2005-05-02 | 2015-12-09 | ジェンザイム・コーポレーション | 神経代謝性疾患のための遺伝子治療 |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| WO2006121960A2 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| CN106729752A (zh) * | 2006-02-08 | 2017-05-31 | 建新公司 | A型尼曼‑皮克病的基因治疗 |
| US9273356B2 (en) | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
| US8324367B2 (en) | 2006-11-03 | 2012-12-04 | Medtronic, Inc. | Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity |
| US9375440B2 (en) | 2006-11-03 | 2016-06-28 | Medtronic, Inc. | Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity |
| US7988668B2 (en) | 2006-11-21 | 2011-08-02 | Medtronic, Inc. | Microsyringe for pre-packaged delivery of pharmaceuticals |
| US7819842B2 (en) | 2006-11-21 | 2010-10-26 | Medtronic, Inc. | Chronically implantable guide tube for repeated intermittent delivery of materials or fluids to targeted tissue sites |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| WO2016100575A1 (en) | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| EP3285788B1 (en) | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN109415730A (zh) | 2016-06-13 | 2019-03-01 | 北卡罗来纳大学教堂山分校 | 优化的cln1基因和表达盒以及它们的应用 |
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| CN112203697A (zh) | 2018-04-13 | 2021-01-08 | 马萨诸塞大学 | 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途 |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| 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 |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| CN113631182B (zh) | 2018-10-10 | 2025-03-21 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
| KR20220022107A (ko) | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| EP4284442A4 (en) * | 2021-02-01 | 2025-04-23 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| ATE386131T1 (de) | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| 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 |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
-
2004
- 2004-04-30 EP EP04760694.2A patent/EP1620133B1/en not_active Expired - Lifetime
- 2004-04-30 EP EP10179332.1A patent/EP2269658B1/en not_active Expired - Lifetime
- 2004-04-30 EP EP18212196.2A patent/EP3513814A1/en not_active Withdrawn
- 2004-04-30 SI SI200432298T patent/SI1620133T1/sl unknown
- 2004-04-30 DK DK04760694.2T patent/DK1620133T3/en active
- 2004-04-30 PT PT47606942T patent/PT1620133E/pt unknown
- 2004-04-30 JP JP2006514239A patent/JP4979067B2/ja not_active Expired - Fee Related
- 2004-04-30 HU HUE04760694A patent/HUE026861T2/en unknown
- 2004-04-30 WO PCT/US2004/013685 patent/WO2004098648A1/en not_active Ceased
- 2004-04-30 PL PL04760694T patent/PL1620133T3/pl unknown
- 2004-04-30 ES ES04760694.2T patent/ES2562490T3/es not_active Expired - Lifetime
-
2005
- 2005-10-27 IL IL171635A patent/IL171635A/en active IP Right Grant
-
2011
- 2011-08-01 JP JP2011168292A patent/JP2011251992A/ja active Pending
-
2013
- 2013-09-24 JP JP2013196398A patent/JP5841574B2/ja not_active Expired - Fee Related
-
2015
- 2015-05-26 JP JP2015106021A patent/JP6170519B2/ja not_active Expired - Fee Related
-
2016
- 2016-03-09 CY CY20161100204T patent/CY1117457T1/el unknown
- 2016-10-20 JP JP2016205651A patent/JP6312771B2/ja not_active Expired - Lifetime
-
2017
- 2017-06-11 IL IL252829A patent/IL252829A0/en unknown
-
2018
- 2018-01-05 JP JP2018000518A patent/JP2018058901A/ja active Pending
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