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1. 发明专利

JP2008013577A Ibudilast for treating neuropathic pain and associated syndrome 审中-公开
标题翻译：用于治疗神经性疼痛和相关综合征的IBUDILAST
公开(公告)号：JP2008013577A
公开(公告)日：2008-01-24
申请号：JP2007234786
申请日：2007-09-10
申请人： Avigen Inc , アビジェン, インコーポレイテッド
发明人： JOHNSON KIRK W , SULZBAUGH LANCE , SHUMILLA JENNIFER
IPC分类号： A61K31/437 , A61P25/02 , A61P25/04 , C07D471/04
CPC分类号： A61K31/4745 , A61K31/437
摘要： PROBLEM TO BE SOLVED: To improve a composition and a method for the treatment of pain, particularly neuropathic pain and associated syndrome, more particularly the treatment of neuropathic pain relating to a specific state such as fibromyalgia. SOLUTION: The invention provides a method for the treatment of mammalian examinee suffered from neuropathic pain. The method comprises a step to select a mammalian examinee suffered from neuropathic pain, and a step to administer the examinee with initial treatment dose of ibudilast effective for achieving the maximum plasma concentration of ibudilast of at least about 125 ng/ml. As a result of the administration, the examinee experiences the reduction of the neuropathic pain. COPYRIGHT: (C)2008,JPO&INPIT
摘要翻译：要解决的问题：改善用于治疗疼痛，特别是神经性疼痛和相关综合征的组合物和方法，更具体地涉及与特定状态如纤维肌痛有关的神经性疼痛的治疗。解决方案：本发明提供了治疗患有神经性疼痛的哺乳动物受试者的方法。该方法包括选择患有神经性疼痛的哺乳动物受试者的步骤，以及用于实现至少约125ng / ml的ibudilast的最大血浆浓度有效的起始治疗剂量的受试者的施用步骤。作为管理的结果，受试者经历神经性疼痛的减少。版权所有（C）2008，JPO＆INPIT

2. 发明专利

JP2011046748A Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulation made therewith 有权
标题翻译：用于ADENO相关病毒药物制剂的药物制剂及其药物制剂
公开(公告)号：JP2011046748A
公开(公告)日：2011-03-10
申请号：JP2010274133
申请日：2010-12-08
申请人： Avigen Inc , アビジェン, インコーポレイテッド
发明人： SISTA HEMA S , ESPINOZA YERO J
IPC分类号： A61K9/00 , A61K35/76 , A61K9/19 , A61K38/48 , A61K47/10 , A61K47/14 , A61K47/26 , A61K47/34 , A61K48/00 , C12N15/864
CPC分类号： C12N15/86 , A61K9/0019 , A61K38/4846 , A61K47/10 , A61K47/26 , A61K48/00 , C12N2750/14143 , C12N2750/14151
摘要： PROBLEM TO BE SOLVED: To provide formulations for storing rAAV (recombinant adeno-associated virus) virions. SOLUTION: There are described a stable pharmaceutical compositions comprising the rAAV virions. The compositions provide protection against loss of rAAV vector genomes and transduceability under conditions such as exposure to cycles of freezing and thawing and storage in glass and polypropylene vials. The compositions comprise the rAAV virions in combination with one or more dihydric alcohols or polyhydric alcohols, and, optionally, a surfactant such as a sorbitan ester. There are also described methods for using the compositions. COPYRIGHT: (C)2011,JPO&INPIT
摘要翻译：待解决的问题：提供用于储存rAAV（重组腺相关病毒）病毒粒子的制剂。解决方案：描述了包含rAAV病毒粒子的稳定的药物组合物。该组合物在诸如暴露于玻璃和聚丙烯小瓶中的冷冻和解冻和储存循环的条件下提供了防止rAAV载体基因组损失和转导性的保护。组合物包含与一种或多种二元醇或多元醇组合的rAAV病毒体，以及任选的表面活性剂如脱水山梨醇酯。还描述了使用组合物的方法。版权所有（C）2011，JPO＆INPIT

3. 发明专利

JP2010273690A Aav virion with decreased immunoreactivity and use therefor 有权
标题翻译： AAV VIRION具有减少的免疫力和使用
公开(公告)号：JP2010273690A
公开(公告)日：2010-12-09
申请号：JP2010196261
申请日：2010-09-01
申请人： Avigen Inc , アビジェン, インコーポレイテッド
发明人： ARBETMAN ALEJANDRA ELENA , COLOSI PETER , LOCHRIE MICHAEL A , SUROSKY RICHARD T
IPC分类号： C12N15/09 , A61K20060101 , A61K35/76 , A61K38/46 , A61K48/00 , C07K1/00 , C07K14/00 , C07K14/015 , C07K17/00 , C12N7/00 , C12N15/00 , C12N15/63 , C12N15/70 , C12N15/74
CPC分类号： A61K48/0066 , A61K38/4846 , A61K48/00 , A61K48/0058 , A61K48/0075 , C07K14/005 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14122 , C12N2750/14143 , C12N2750/14162 , C12N2830/008 , C12N2830/85 , C12Y304/21022
摘要： PROBLEM TO BE SOLVED: To provide an AAV variant with decreased immunoreactivity. SOLUTION: There is disclosed a method for preparing recombinant AAV virion with decreased immunoreactivity and use of the same. The recombinant AAV virion includes mutated capsid proteins or is derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2. There are provided, in one embodiment, a method for effective delivery of heterologous nucleic acid molecule (HNA) to cells or tissues of a vertebrate subject (for example, a mammalian) using a recombinant AAV virion, and an AAV vector for using in the method. COPYRIGHT: (C)2011,JPO&INPIT
摘要翻译：要解决的问题：提供具有降低的免疫反应性的AAV变体。解决方案：公开了一种制备具有降低的免疫反应性的重组AAV病毒粒子的方法及其用途。重组AAV病毒粒子包括突变的衣壳蛋白或衍生自非灵长类哺乳动物AAV血清型和相对于AAV-2显示降低的免疫反应性的分离物。在一个实施方案中，提供了使用重组AAV病毒体将异源核酸分子（HNA）有效递送至脊椎动物受试者（例如哺乳动物）的细胞或组织的方法，以及用于方法。版权所有（C）2011，JPO＆INPIT

4. 发明专利

JP2006298926A Convection-enhanced delivery of Aav vector 审中-公开
标题翻译：对流增强AAV矢量传递
公开(公告)号：JP2006298926A
公开(公告)日：2006-11-02
申请号：JP2006140195
申请日：2006-05-19
申请人： Avigen Inc , Univ California , アビジェン, インコーポレイテッド , ザリージェンツオブザユニバーシティオブカリフォルニア
发明人： BANKIEWICZ KRYS , CUNNINGHAM JANET , EBERLING JAMIE L
IPC分类号： A61K48/00 , C12N15/09 , A61K31/198 , A61K31/711 , A61K35/76 , A61K38/00 , A61K38/43 , A61K38/45 , A61K38/51 , A61K47/48 , A61K51/00 , A61K51/04 , A61P25/16
CPC分类号： A61K31/198 , A01N63/00 , A61K9/5184 , A61K35/761 , A61K38/45 , A61K38/51 , A61K45/06 , A61K47/6901 , A61K48/00 , A61K48/0025 , A61K48/0075 , A61K51/00 , A61K51/0402 , C12N7/00 , C12N15/63 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12Y401/01028
摘要： PROBLEM TO BE SOLVED: To provide a method of delivering a rAAV (recombinant adeno-associated virus) virion to the CNS (central nervous system) of a test object. SOLUTION: The method of delivering the recombinant AAV virion to the test object involves a step for administering the rAAV virion through CED (convection-enhanced delivery) into the CNS of the test object, wherein the rAAV virion contains a nucleic acid sequence encoding a polypeptide for treatment. The CED is carried out for example by using either of an osmotic pressure pump or an injection pump. COPYRIGHT: (C)2007,JPO&INPIT
摘要翻译：待解决的问题：提供将rAAV（重组腺相关病毒）病毒粒子递送到测试对象的CNS（中枢神经系统）的方法。解决方案：将重组AAV病毒粒子递送到测试对象的方法包括通过CED（对流增强递送）将rAAV病毒体施用到测试对象的CNS中的步骤，其中rAAV病毒粒子含有核酸序列编码多肽进行治疗。 CED例如通过使用渗透压泵或注射泵中的任一种进行。版权所有（C）2007，JPO＆INPIT

5. 发明专利

JP2006223315A HIGH EFFICIENCY HELPER SYSTEM FOR AAV VECTOR PRODUCTION 审中-公开
公开(公告)号：JP2006223315A
公开(公告)日：2006-08-31
申请号：JP2006152816
申请日：2006-05-31
申请人： AVIGEN INC
发明人： NATSOULIS GEORGES
IPC分类号： C12N15/09 , C07K14/015 , C12N5/10 , C12N7/04 , C12N15/864
摘要： PROBLEM TO BE SOLVED: To provide an improved adeno-associated virus (AAV) helper function structure which can be expressed in host cells at effective levels. SOLUTION: Novel nucleic acid molecules are provided having AAV coding regions that are capable of expressing necessary AAV functions to complement an AAV vector in the production of recombinant adeno-associated virus (rAAV) virions. The molecules feature a nucleotide sequence that is substantially homologous to an AAV p5 promoter region, wherein the p5 promoter region is situated in the molecules in a site that is other than its natural position relative to the AAV rep coding region in the wild-type AAV genome. AAV helper function structures, novel AAV packaging cells, AAV producer cells, methods of producing enhanced levels of rAAV virions using the AAV helper structures and methods for producing rAAV virions without the concomitant production of significant levels of wild-type AAV are also provided. COPYRIGHT: (C)2006,JPO&NCIPI

6. 发明专利

JP2006075171A Accessory functions for use in recombinant aav virion production 审中-公开
标题翻译：用于重组AAV病毒生产的附件功能
公开(公告)号：JP2006075171A
公开(公告)日：2006-03-23
申请号：JP2005306447
申请日：2005-10-20
申请人： Avigen Inc , アビジェン, インコーポレイテッド
发明人： COLOSI PETER C
IPC分类号： C12N15/09
摘要： PROBLEM TO BE SOLVED: To provide accessory function systems capable of producing rAAV virion particles in commercially significant level without generating significant levels of infectious adenovirus virions or other contaminating by-products. SOLUTION: Disclosed is a nucleic acid molecule providing one or more accessory functions in a level supporting efficient recombinant AAV (rAAV) virion production in a host cell, wherein the molecule is featured by being selected from the group consisting of (i) a sequence providing an adenovirus VA RNA, (ii) an adenovirus E4 ORF6 coding region, (iii) an adenovirus E2a 72 kD coding region and an optional combination of (i), (ii) and (iii). COPYRIGHT: (C)2006,JPO&NCIPI
摘要翻译：要解决的问题：提供能够以商业上显着水平生产rAAV病毒颗粒的辅助功能系统，而不产生显着水平的感染性腺病毒病毒颗粒或其他污染副产物。解决方案：公开了提供在宿主细胞中支持有效重组AAV（rAAV）病毒粒子产生的水平的一个或多个辅助功能的核酸分子，其中该分子的特征在于选自（i）提供腺病毒VA RNA的序列，（ii）腺病毒E4 ORF6编码区，（iii）腺病毒E2a 72kD编码区和（i），（ii）和（iii）的任选组合。版权所有（C）2006，JPO＆NCIPI

7. 发明申请

US20020115189A1 High-efficiency AAV helper functions 审中-公开
标题翻译：高效AAV助手功能
公开(公告)号：US20020115189A1
公开(公告)日：2002-08-22
申请号：US10026389
申请日：2001-12-18
申请人： Avigen, Inc.
发明人： Georges Natsoulis , Gary Kurtzman , Peter Colosi
IPC分类号： C12N007/00 , C12N015/86 , C12N005/00
CPC分类号： C12N15/86 , C07K14/005 , C07K2319/00 , C12N7/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152 , C12N2750/14162 , C12N2800/30 , C12N2840/20
摘要： The present invention provides methods and compositions for producing high titer preparations of recombinant AAV (nullrAAVnull) virions. The compositions of the present invention include AAV helper function systems and host cells. The present invention also includes methods of using AAV helper function vectors that effect the production of only small amounts of the long forms of Rep protein, and rAAV virions produced by such methods.
摘要翻译：本发明提供了用于生产重组AAV（“rAAV”）病毒粒子的高效价制剂的方法和组合物。本发明的组合物包括AAV辅助功能体系和宿主细胞。本发明还包括使用影响仅产生少量Rep形式的Rep蛋白的AAV辅助功能载体的方法和通过这些方法产生的rAAV病毒体。

8. 发明申请

US20040018627A1 High-efficiency AAV helper functions 审中-公开
标题翻译：高效AAV助手功能
公开(公告)号：US20040018627A1
公开(公告)日：2004-01-29
申请号：US10631403
申请日：2003-07-30
申请人： Avigen, Inc.
发明人： Georges Natsoulis , Gary Kurtzman , Peter Colosi
IPC分类号： C12N015/861 , C12N007/00
CPC分类号： C12N15/86 , C07K14/005 , C07K2319/00 , C12N7/00 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152 , C12N2750/14162 , C12N2800/30 , C12N2840/20
摘要： The present invention provides methods and compositions for producing high titer preparations of recombinant AAV (nullrAAVnull) virions. The compositions of the present invention include AAV helper function systems and host cells. The present invention also includes methods of using AAV helper function vectors that effect the production of only small amounts of the long forms of Rep protein, and rAAV virions produced by such methods.
摘要翻译：本发明提供了用于生产重组AAV（“rAAV”）病毒粒子的高效价制剂的方法和组合物。本发明的组合物包括AAV辅助功能体系和宿主细胞。本发明还包括使用影响仅产生少量Rep形式的Rep蛋白的AAV辅助功能载体的方法和通过这些方法产生的rAAV病毒体。

9. 发明授权

US6391858B2 Methods for delivering DNA to the bloodstream using recombinant adeno-associated virus vectors 有权
标题翻译：使用重组腺相关病毒载体将DNA递送到血流的方法
公开(公告)号：US6391858B2
公开(公告)日：2002-05-21
申请号：US75573401
申请日：2001-01-04
申请人： AVIGEN INC
发明人： PODSAKOFF GREGORY M , KURTZMAN GARY J
IPC分类号： A61K38/00 , A61K38/18 , A61K38/47 , A61K48/00 , C07K14/505 , C12N5/08 , C12N7/01 , C12N9/24 , C12N15/864 , C12N15/00 , C12P21/06
CPC分类号： C12N15/86 , A01K2217/05 , A61K38/1816 , A61K38/47 , A61K48/00 , C07K14/505 , C12N9/2471 , C12N2750/14143 , C12N2830/42 , C12N2840/44 , C12Y302/0102 , C12Y302/01023
摘要： The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.
摘要翻译：公开了使用重组腺相关病毒（AAV）病毒颗粒将DNA分子递送至肌肉细胞和组织。本发明允许将重组AAV病毒体直接，体内注射到肌肉组织中，例如通过肌内注射，以及肌肉细胞的体外转导，随后可将其导入受试者进行治疗。本发明提供所传递的基因的持续高水平表达以及用于体内分泌来自转导的肌细胞的治疗性蛋白质，从而实现全身递送。

10. 发明申请

US20040258665A1 Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith 有权
公开(公告)号：US20040258665A1
公开(公告)日：2004-12-23
申请号：US10862036
申请日：2004-06-04
申请人： Avigen, Inc.
发明人： Hema S. Sista , Yero J. Espinoza
IPC分类号： A61K048/00 , A61K031/045
CPC分类号： A61K47/26 , A61K9/0019 , A61K38/4846 , A61K47/10 , A61K48/00 , A61K48/0008 , A61K48/0091 , C12N15/86 , C12N2750/14143 , C12N2750/14151
摘要： Stable pharmaceutical compositions comprising recombinant adeno-associated virus (AAV) virions are described. The compositions provide protection against loss of recombinant AAV vector genomes and transduceability under conditions such as exposure to cycles of freezing and thawing and storage in glass or polypropylene vials. The compositions comprise recombinant AAV virions in combination with one or more dihydric or polyhydric alcohols, and, optionally, a detergent, such as a sorbitan ester. Also described are methods of using the compositions.

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