JP2009526067A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2009526067A5 JP2009526067A5 JP2008554342A JP2008554342A JP2009526067A5 JP 2009526067 A5 JP2009526067 A5 JP 2009526067A5 JP 2008554342 A JP2008554342 A JP 2008554342A JP 2008554342 A JP2008554342 A JP 2008554342A JP 2009526067 A5 JP2009526067 A5 JP 2009526067A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- aav2
- vector
- administered
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 25
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 12
- 239000013607 AAV vector Substances 0.000 claims 8
- 239000013603 viral vector Substances 0.000 claims 8
- 241000124008 Mammalia Species 0.000 claims 7
- 108700019146 Transgenes Proteins 0.000 claims 7
- 210000005171 mammalian brain Anatomy 0.000 claims 5
- 229920001184 polypeptide Polymers 0.000 claims 5
- 102000004196 processed proteins & peptides Human genes 0.000 claims 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 102000004169 proteins and genes Human genes 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 5
- 210000001638 cerebellum Anatomy 0.000 claims 4
- 210000001259 mesencephalon Anatomy 0.000 claims 4
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 3
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 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
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 210000000133 brain stem Anatomy 0.000 claims 2
- 210000003591 cerebellar nuclei Anatomy 0.000 claims 2
- 210000003710 cerebral cortex Anatomy 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 210000001652 frontal lobe Anatomy 0.000 claims 2
- 210000001320 hippocampus Anatomy 0.000 claims 2
- 210000003016 hypothalamus Anatomy 0.000 claims 2
- 230000002163 immunogen Effects 0.000 claims 2
- 210000001577 neostriatum Anatomy 0.000 claims 2
- 210000000869 occipital lobe Anatomy 0.000 claims 2
- 210000001152 parietal lobe Anatomy 0.000 claims 2
- 210000002975 pon Anatomy 0.000 claims 2
- 210000003478 temporal lobe Anatomy 0.000 claims 2
- 210000001103 thalamus Anatomy 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 208000000609 Pick Disease of the Brain Diseases 0.000 claims 1
- 208000024571 Pick disease Diseases 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 210000005228 liver tissue Anatomy 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US77162806P | 2006-02-08 | 2006-02-08 | |
| US60/771,628 | 2006-02-08 | ||
| US77236006P | 2006-02-09 | 2006-02-09 | |
| US60/772,360 | 2006-02-09 | ||
| PCT/US2007/003388 WO2007092563A2 (en) | 2006-02-08 | 2007-02-08 | Gene therapy for niemann-pick disease type a |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013092907A Division JP5881641B2 (ja) | 2006-02-08 | 2013-04-25 | ニーマンピック病ａ型に対する遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2009526067A JP2009526067A (ja) | 2009-07-16 |
| JP2009526067A5 true JP2009526067A5 (https=) | 2010-04-08 |
| JP5706602B2 JP5706602B2 (ja) | 2015-04-22 |
Family
ID=38345806
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008554342A Active JP5706602B2 (ja) | 2006-02-08 | 2007-02-08 | ニーマンピック病ａ型に対する遺伝子治療 |
| JP2013092907A Active JP5881641B2 (ja) | 2006-02-08 | 2013-04-25 | ニーマンピック病ａ型に対する遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013092907A Active JP5881641B2 (ja) | 2006-02-08 | 2013-04-25 | ニーマンピック病ａ型に対する遺伝子治療 |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US20090117156A1 (https=) |
| EP (2) | EP1986661B1 (https=) |
| JP (2) | JP5706602B2 (https=) |
| AR (1) | AR059371A1 (https=) |
| BR (1) | BRPI0707590A2 (https=) |
| CY (1) | CY1121380T1 (https=) |
| DK (1) | DK1986661T3 (https=) |
| ES (1) | ES2700048T3 (https=) |
| HU (1) | HUE041928T2 (https=) |
| IL (2) | IL193165B (https=) |
| LT (1) | LT1986661T (https=) |
| MX (3) | MX360595B (https=) |
| PL (1) | PL1986661T3 (https=) |
| PT (1) | PT1986661T (https=) |
| SI (1) | SI1986661T1 (https=) |
| WO (1) | WO2007092563A2 (https=) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CA2606490C (en) | 2005-05-02 | 2018-02-06 | Genzyme Corporation | Gene therapy for spinal cord disorders |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP2561073B1 (en) * | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 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 |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| US11007280B2 (en) * | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| CA3038292A1 (en) | 2016-09-28 | 2018-04-05 | Cohbar, Inc. | Therapeutic mots-c related peptides |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| CA3041548A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
| US11117930B2 (en) | 2017-02-23 | 2021-09-14 | Adrx, Inc. | Peptide inhibitors of transcription factor aggregation |
| EP3634978A1 (en) | 2017-06-07 | 2020-04-15 | Adrx, Inc. | Tau aggregation inhibitors |
| CA3068010A1 (en) | 2017-06-20 | 2018-12-27 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| SG11202107761SA (en) | 2019-01-28 | 2021-08-30 | Cohbar Inc | Therapeutic peptides |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| PE20212082A1 (es) * | 2019-04-24 | 2021-10-28 | Univ Oregon Health & Science | Secuencia promotora y productos y usos relacionados de la misma |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2023532923A (ja) * | 2020-06-30 | 2023-08-01 | チルドレンズ ナショナル メディカル センター | 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用 |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| 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 (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL154598B (nl) | 1970-11-10 | 1977-09-15 | Organon Nv | Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking. |
| US3817837A (en) | 1971-05-14 | 1974-06-18 | Syva Corp | Enzyme amplification assay |
| US3939350A (en) | 1974-04-29 | 1976-02-17 | Board Of Trustees Of The Leland Stanford Junior University | Fluorescent immunoassay employing total reflection for activation |
| US3996345A (en) | 1974-08-12 | 1976-12-07 | Syva Company | Fluorescence quenching with immunological pairs in immunoassays |
| US4275149A (en) | 1978-11-24 | 1981-06-23 | Syva Company | Macromolecular environment control in specific receptor assays |
| US4277437A (en) | 1978-04-05 | 1981-07-07 | Syva Company | Kit for carrying out chemically induced fluorescence immunoassay |
| US4366241A (en) | 1980-08-07 | 1982-12-28 | Syva Company | Concentrating zone method in heterogeneous immunoassays |
| DE3424893A1 (de) | 1984-07-06 | 1986-02-06 | Agfa-Gevaert Ag, 5090 Leverkusen | Photographisches silberhalogenidaufzeichnungsmaterial |
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US6040138A (en) | 1995-09-15 | 2000-03-21 | Affymetrix, Inc. | Expression monitoring by hybridization to high density oligonucleotide arrays |
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| US5412087A (en) | 1992-04-24 | 1995-05-02 | Affymax Technologies N.V. | Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces |
| US6114114A (en) | 1992-07-17 | 2000-09-05 | Incyte Pharmaceuticals, Inc. | Comparative gene transcript analysis |
| EP0705344B8 (en) | 1993-06-24 | 2006-05-10 | Advec Inc. | Adenovirus vectors for gene therapy |
| AU687117B2 (en) | 1993-10-25 | 1998-02-19 | Canji, Inc. | Recombinant adenoviral vector and methods of use |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5631734A (en) | 1994-02-10 | 1997-05-20 | Affymetrix, Inc. | Method and apparatus for detection of fluorescently labeled materials |
| US5578832A (en) | 1994-09-02 | 1996-11-26 | Affymetrix, Inc. | Method and apparatus for imaging a sample on a device |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| ATE386131T1 (de) | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| US5795716A (en) | 1994-10-21 | 1998-08-18 | Chee; Mark S. | Computer-aided visualization and analysis system for sequence evaluation |
| IL135578A0 (en) | 1997-10-29 | 2001-05-20 | Genzyme Corp | Compositions and methods for treating lysosomal storage disease |
| 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 |
| ES2284545T3 (es) | 1999-11-16 | 2007-11-16 | Genzyme Corporation | Vectores y transgenes con elementos reguladores para la administracion de genes del higado. |
| US6582692B1 (en) * | 1999-11-17 | 2003-06-24 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| AU2001236713A1 (en) * | 2000-02-17 | 2001-08-27 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases |
| WO2001060414A2 (en) * | 2000-02-17 | 2001-08-23 | Genzyme Corporation | Genetic modification of the lung as a portal for gene delivery |
| US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| CA2361462A1 (en) * | 2001-11-07 | 2003-05-08 | Katherine A. High | Induction of tolerance to a therapeutic polypeptide |
| US20060057110A1 (en) * | 2002-06-14 | 2006-03-16 | Yeda Research And Development Co. Ltd. | Antigen-presenting cells for neuroprotection and nerve regeneration |
| AU2003265855A1 (en) * | 2002-08-28 | 2004-03-19 | University Of Florida | Modified aav |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
-
2007
- 2007-02-08 AR ARP070100528A patent/AR059371A1/es not_active Application Discontinuation
- 2007-02-08 BR BRPI0707590-1A patent/BRPI0707590A2/pt not_active IP Right Cessation
- 2007-02-08 HU HUE07763560A patent/HUE041928T2/hu unknown
- 2007-02-08 EP EP07763560.5A patent/EP1986661B1/en active Active
- 2007-02-08 JP JP2008554342A patent/JP5706602B2/ja active Active
- 2007-02-08 WO PCT/US2007/003388 patent/WO2007092563A2/en not_active Ceased
- 2007-02-08 PL PL07763560T patent/PL1986661T3/pl unknown
- 2007-02-08 DK DK07763560.5T patent/DK1986661T3/da active
- 2007-02-08 MX MX2014005624A patent/MX360595B/es unknown
- 2007-02-08 EP EP18190420.2A patent/EP3456331B1/en active Active
- 2007-02-08 LT LTEP07763560.5T patent/LT1986661T/lt unknown
- 2007-02-08 SI SI200732075T patent/SI1986661T1/sl unknown
- 2007-02-08 ES ES07763560T patent/ES2700048T3/es active Active
- 2007-02-08 PT PT07763560T patent/PT1986661T/pt unknown
-
2008
- 2008-07-31 IL IL193165A patent/IL193165B/en not_active IP Right Cessation
- 2008-08-07 US US12/187,844 patent/US20090117156A1/en not_active Abandoned
- 2008-08-07 MX MX2018008355A patent/MX2018008355A/es unknown
- 2008-08-07 MX MX2018013684A patent/MX2018013684A/es unknown
-
2013
- 2013-04-25 JP JP2013092907A patent/JP5881641B2/ja active Active
-
2018
- 2018-04-09 IL IL258587A patent/IL258587A/en unknown
- 2018-11-28 CY CY20181101265T patent/CY1121380T1/el unknown
-
2019
- 2019-08-19 US US16/544,735 patent/US20200080066A1/en not_active Abandoned
-
2023
- 2023-03-08 US US18/119,201 patent/US20230287366A1/en not_active Abandoned