JP2009526066A5 - - Google Patents
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- JP2009526066A5 JP2009526066A5 JP2008554340A JP2008554340A JP2009526066A5 JP 2009526066 A5 JP2009526066 A5 JP 2009526066A5 JP 2008554340 A JP2008554340 A JP 2008554340A JP 2008554340 A JP2008554340 A JP 2008554340A JP 2009526066 A5 JP2009526066 A5 JP 2009526066A5
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- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- drug
- patient
- agent
- rate
- 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.)
- Pending
Links
- 239000008194 pharmaceutical composition Substances 0.000 claims 58
- 239000003814 drug Substances 0.000 claims 38
- 229940079593 drug Drugs 0.000 claims 27
- 230000007306 turnover Effects 0.000 claims 15
- 239000003795 chemical substances by application Substances 0.000 claims 14
- 210000004556 brain Anatomy 0.000 claims 10
- 210000003140 lateral ventricle Anatomy 0.000 claims 5
- 239000002872 contrast media Substances 0.000 claims 4
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 3
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 3
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 2
- 210000002966 serum Anatomy 0.000 claims 2
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 102000002268 Hexosaminidases Human genes 0.000 claims 1
- 108010000540 Hexosaminidases Proteins 0.000 claims 1
- 102000004627 Iduronidase Human genes 0.000 claims 1
- 108010003381 Iduronidase Proteins 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 201000000791 Niemann-Pick disease type B Diseases 0.000 claims 1
- 102000011971 Sphingomyelin Phosphodiesterase Human genes 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 208000022292 Tay-Sachs disease Diseases 0.000 claims 1
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 230000003444 anaesthetic effect Effects 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 210000004055 fourth ventricle Anatomy 0.000 claims 1
- 208000007345 glycogen storage disease Diseases 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 239000011148 porous material Substances 0.000 claims 1
- 230000002285 radioactive effect Effects 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 210000002330 subarachnoid space Anatomy 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
- 210000000211 third ventricle Anatomy 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US77145106P | 2006-02-09 | 2006-02-09 | |
| PCT/US2007/003382 WO2007095056A2 (en) | 2006-02-09 | 2007-02-08 | Slow intraventricular delivery |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013171131A Division JP6129685B2 (ja) | 2006-02-09 | 2013-08-21 | 遅速性脳室内送達 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2009526066A JP2009526066A (ja) | 2009-07-16 |
| JP2009526066A5 true JP2009526066A5 (https=) | 2010-04-08 |
Family
ID=38372004
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008554340A Pending JP2009526066A (ja) | 2006-02-09 | 2007-02-08 | 遅速性脳室内送達 |
| JP2013171131A Active JP6129685B2 (ja) | 2006-02-09 | 2013-08-21 | 遅速性脳室内送達 |
| JP2015188744A Active JP6408970B2 (ja) | 2006-02-09 | 2015-09-25 | 遅速性脳室内送達 |
| JP2017152871A Active JP6617125B2 (ja) | 2006-02-09 | 2017-08-08 | 遅速性脳室内送達 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013171131A Active JP6129685B2 (ja) | 2006-02-09 | 2013-08-21 | 遅速性脳室内送達 |
| JP2015188744A Active JP6408970B2 (ja) | 2006-02-09 | 2015-09-25 | 遅速性脳室内送達 |
| JP2017152871A Active JP6617125B2 (ja) | 2006-02-09 | 2017-08-08 | 遅速性脳室内送達 |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US20090123451A1 (https=) |
| EP (2) | EP1988823B1 (https=) |
| JP (4) | JP2009526066A (https=) |
| CN (1) | CN101404927A (https=) |
| AR (1) | AR059372A1 (https=) |
| BR (1) | BRPI0707900A2 (https=) |
| CA (2) | CA3168881C (https=) |
| CY (1) | CY1121822T1 (https=) |
| DK (1) | DK1988823T3 (https=) |
| ES (2) | ES2697502T3 (https=) |
| HU (1) | HUE040490T2 (https=) |
| IL (2) | IL193242A (https=) |
| LT (1) | LT1988823T (https=) |
| MX (4) | MX392030B (https=) |
| PL (1) | PL1988823T3 (https=) |
| PT (1) | PT1988823T (https=) |
| RU (1) | RU2452368C2 (https=) |
| SI (1) | SI1988823T1 (https=) |
| WO (1) | WO2007095056A2 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| DK1988823T3 (en) * | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| EP2167071B1 (en) * | 2007-06-13 | 2020-03-18 | Wayne State University Board Of Governors | A baclofen solution for low-volume therapeutic delivery |
| CN102549152B (zh) | 2009-07-31 | 2015-11-25 | 帕克斯瓦克斯股份有限公司 | 基于腺病毒的载体 |
| SI3998078T1 (sl) | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| RS62620B1 (sr) | 2010-06-25 | 2021-12-31 | Shire Human Genetic Therapies | Metode i kompozicije za isporuku cns akrilsulfataze a |
| RS61055B1 (sr) | 2012-06-19 | 2020-12-31 | Univ Florida | Kompozicije i postupci za lečenje bolesti |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
Family Cites Families (32)
| 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 |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5919676A (en) | 1993-06-24 | 1999-07-06 | Advec, Inc. | Adenoviral vector system comprising Cre-loxP recombination |
| AU7676894A (en) * | 1993-08-27 | 1995-03-21 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Convection-enhanced drug delivery |
| CA2189067A1 (en) | 1994-04-28 | 1995-11-09 | Gary J. Nabel | Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line |
| DE69628744D1 (de) | 1995-04-17 | 2003-07-24 | Univ Texas System Austin Board | Adenovirus helfervirus system |
| SI0833934T2 (sl) | 1995-06-15 | 2013-04-30 | Crucell Holland B.V. | Pakirni sistemi za humani rekombinantni adenovirus za uporabo v genski terapiji |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| CA2239366A1 (en) | 1996-01-05 | 1997-07-17 | Genetic Therapy, Inc. | Recombinase-mediated generation of adenoviral vectors |
| US5735814A (en) * | 1996-04-30 | 1998-04-07 | Medtronic, Inc. | Techniques of treating neurodegenerative disorders by brain infusion |
| JP4001925B2 (ja) | 1996-09-13 | 2007-10-31 | トランスカーヨティック セラピーズ インク. | α―ガラクトシダーゼA欠損症の治療 |
| US7033598B2 (en) * | 1996-11-19 | 2006-04-25 | Intrabrain International N.V. | Methods and apparatus for enhanced and controlled delivery of a biologically active agent into the central nervous system of a mammal |
| US6042579A (en) * | 1997-04-30 | 2000-03-28 | Medtronic, Inc. | Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| WO1999057296A1 (en) | 1998-05-01 | 1999-11-11 | Genzyme Corporation | Partially deleted adenoviral vectors |
| US20060014166A1 (en) | 2004-01-27 | 2006-01-19 | Yossi Cohen | Novel nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of endometriosis |
| US6702945B2 (en) | 2000-12-28 | 2004-03-09 | Exxonmobil Research And Engineering Company | Ionic membranes for organic sulfur separation from liquid hydrocarbon solutions |
| US6689756B2 (en) * | 2001-03-02 | 2004-02-10 | Integra Lifesciences Corporation | Treatment of neurological disease |
| AU2002357805A1 (en) * | 2001-12-07 | 2003-06-23 | Neuron Therapeutics | Protection of neurological tissue by direct cns perfusion cooling |
| US7923032B2 (en) * | 2002-11-26 | 2011-04-12 | Seacoast Neuroscience, Inc. | Buoyant polymer particles for delivery of therapeutic agents to the central nervous system |
| RU2253486C2 (ru) * | 2003-03-03 | 2005-06-10 | Ростовский научно-исследовательский онкологический институт МЗ РФ | Способ химиотерапии опухолей центральной нервной системы |
| CA2518407A1 (en) * | 2003-03-12 | 2004-09-23 | Samaritan Pharmaceuticals, Inc. | Animal model simulating neurologic disease |
| US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| CA2525236C (en) * | 2003-06-20 | 2015-03-24 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| US20080233655A1 (en) * | 2004-03-31 | 2008-09-25 | Children, Youth And Women's Health Service | Screening For Lysosomal Storage Disease Status |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| AR059088A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una proteina para esclerosis lateral amiotrofica |
| DK1988823T3 (en) | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
-
2007
- 2007-02-08 DK DK07750236.7T patent/DK1988823T3/en active
- 2007-02-08 EP EP07750236.7A patent/EP1988823B1/en active Active
- 2007-02-08 PT PT07750236T patent/PT1988823T/pt unknown
- 2007-02-08 AR ARP070100529A patent/AR059372A1/es not_active Application Discontinuation
- 2007-02-08 CA CA3168881A patent/CA3168881C/en active Active
- 2007-02-08 EP EP18188582.3A patent/EP3459441B1/en active Active
- 2007-02-08 PL PL07750236T patent/PL1988823T3/pl unknown
- 2007-02-08 WO PCT/US2007/003382 patent/WO2007095056A2/en not_active Ceased
- 2007-02-08 RU RU2008136196/14A patent/RU2452368C2/ru not_active IP Right Cessation
- 2007-02-08 SI SI200732069T patent/SI1988823T1/sl unknown
- 2007-02-08 MX MX2018015511A patent/MX392030B/es unknown
- 2007-02-08 MX MX2008010125A patent/MX358492B/es active IP Right Grant
- 2007-02-08 ES ES07750236T patent/ES2697502T3/es active Active
- 2007-02-08 LT LTEP07750236.7T patent/LT1988823T/lt unknown
- 2007-02-08 ES ES18188582T patent/ES3054134T3/es active Active
- 2007-02-08 CN CNA2007800098972A patent/CN101404927A/zh active Pending
- 2007-02-08 BR BRPI0707900-1A patent/BRPI0707900A2/pt not_active IP Right Cessation
- 2007-02-08 CA CA2641359A patent/CA2641359C/en active Active
- 2007-02-08 JP JP2008554340A patent/JP2009526066A/ja active Pending
- 2007-02-08 HU HUE07750236A patent/HUE040490T2/hu unknown
- 2007-02-08 MX MX2013013333A patent/MX361816B/es unknown
-
2008
- 2008-08-05 IL IL193242A patent/IL193242A/en not_active IP Right Cessation
- 2008-08-07 US US12/187,896 patent/US20090123451A1/en not_active Abandoned
- 2008-08-07 MX MX2022005303A patent/MX2022005303A/es unknown
-
2013
- 2013-08-21 JP JP2013171131A patent/JP6129685B2/ja active Active
-
2015
- 2015-09-25 JP JP2015188744A patent/JP6408970B2/ja active Active
-
2016
- 2016-10-25 IL IL248492A patent/IL248492A0/en unknown
-
2017
- 2017-08-08 JP JP2017152871A patent/JP6617125B2/ja active Active
-
2018
- 2018-11-15 CY CY20181101209T patent/CY1121822T1/el unknown
-
2019
- 2019-02-21 US US16/281,961 patent/US11253485B2/en active Active
-
2022
- 2022-01-10 US US17/572,330 patent/US20220370348A1/en not_active Abandoned
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