JP2009526067A5 - - Google Patents
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- 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) | EP3456331B1 (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 (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL1879623T3 (pl) | 2005-05-02 | 2013-03-29 | Genzyme Corp | Terapia genowa zaburzeń rdzenia kręgowego |
| EP1879624B1 (en) | 2005-05-02 | 2011-09-21 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2826860B1 (en) * | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| WO2012145646A1 (en) * | 2011-04-20 | 2012-10-26 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis |
| 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 |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| EP3270944B1 (en) * | 2015-03-17 | 2019-10-23 | 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 |
| IL305449B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
| TW201819398A (zh) | 2016-09-28 | 2018-06-01 | 美商寇峇有限公司 | 治療性肽 |
| EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| EP3534892A4 (en) * | 2016-11-04 | 2020-05-27 | The Children's Hospital of Philadelphia | GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES |
| WO2018156892A1 (en) | 2017-02-23 | 2018-08-30 | Adrx, Inc. | Peptide inhibitors of transcription factor aggregation |
| CN110997693A (zh) | 2017-06-07 | 2020-04-10 | 阿德克斯公司 | τ聚集抑制剂 |
| US12116577B2 (en) | 2017-06-20 | 2024-10-15 | 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 |
| 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 |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| EP3917944A1 (en) | 2019-01-28 | 2021-12-08 | Cohbar Inc. | Therapeutic peptides |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| MX2021012578A (es) * | 2019-04-24 | 2021-12-10 | Univ Oregon Health & Science | Secuencia promotora y productos y usos relacionados de la misma. |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| KR20230031329A (ko) * | 2020-06-30 | 2023-03-07 | 칠드런스 내셔널 메디컬 센터 | 골격 근섬유 복구를 개선하기 위한 재조합 인간 산성 스핑고미엘리나제의 용도 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| JP2024506860A (ja) * | 2021-02-01 | 2024-02-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ニーマンピック病ａ型を治療するための組成物及び方法 |
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 |
| US6040138A (en) | 1995-09-15 | 2000-03-21 | Affymetrix, Inc. | Expression monitoring by hybridization to high density oligonucleotide arrays |
| 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 |
| 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 |
| ES2249761T3 (es) | 1993-06-24 | 2006-04-01 | Advec Inc. | Vectores de adenovirus para terapia genica. |
| EP1637608B1 (en) | 1993-10-25 | 2009-07-22 | CANJI, Inc. | Recombinant adenoviral vector and methods of use |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US5578832A (en) | 1994-09-02 | 1996-11-26 | Affymetrix, Inc. | Method and apparatus for imaging a sample on a device |
| US5631734A (en) | 1994-02-10 | 1997-05-20 | Affymetrix, Inc. | Method and apparatus for detection of fluorescently labeled materials |
| 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 |
| 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. |
| AU1775901A (en) * | 1999-11-17 | 2001-05-30 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| CA2400634A1 (en) * | 2000-02-17 | 2001-08-23 | Chester Li | Genetic modification of the lung as a portal for gene delivery |
| US20010031741A1 (en) * | 2000-02-17 | 2001-10-18 | Robin Ziegler | Methods for treatment of lysosomal storage diseases |
| 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 |
| WO2003105750A2 (en) * | 2002-06-14 | 2003-12-24 | 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 JP JP2008554342A patent/JP5706602B2/ja active Active
- 2007-02-08 WO PCT/US2007/003388 patent/WO2007092563A2/en not_active Ceased
- 2007-02-08 EP EP18190420.2A patent/EP3456331B1/en active Active
- 2007-02-08 DK DK07763560.5T patent/DK1986661T3/da active
- 2007-02-08 PT PT07763560T patent/PT1986661T/pt unknown
- 2007-02-08 PL PL07763560T patent/PL1986661T3/pl unknown
- 2007-02-08 BR BRPI0707590-1A patent/BRPI0707590A2/pt not_active IP Right Cessation
- 2007-02-08 MX MX2014005624A patent/MX360595B/es unknown
- 2007-02-08 AR ARP070100528A patent/AR059371A1/es not_active Application Discontinuation
- 2007-02-08 HU HUE07763560A patent/HUE041928T2/hu unknown
- 2007-02-08 EP EP07763560.5A patent/EP1986661B1/en active Active
- 2007-02-08 LT LTEP07763560.5T patent/LT1986661T/lt unknown
- 2007-02-08 ES ES07763560T patent/ES2700048T3/es active Active
- 2007-02-08 SI SI200732075T patent/SI1986661T1/sl unknown
-
2008
- 2008-07-31 IL IL193165A patent/IL193165B/en not_active IP Right Cessation
- 2008-08-07 MX MX2018013684A patent/MX2018013684A/es unknown
- 2008-08-07 MX MX2018008355A patent/MX2018008355A/es unknown
- 2008-08-07 US US12/187,844 patent/US20090117156A1/en not_active Abandoned
-
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
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2023
- 2023-03-08 US US18/119,201 patent/US20230287366A1/en not_active Abandoned