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1. 发明申请

WO2016170484A1 RNA-GUIDED GENE EDITING SYSTEM AND USES THEREOF 审中-公开
标题翻译： RNA指导基因编码系统及其用途
公开(公告)号：WO2016170484A1
公开(公告)日：2016-10-27
申请号：PCT/IB2016/052243
申请日：2016-04-20
申请人： NOVARTIS AG , LOEW, Andreas , HUEBNER, Anette
发明人： LOEW, Andreas , HUEBNER, Anette
IPC分类号： C12N15/90 , C12N15/10 , C12N15/63 , C12N5/00
CPC分类号： C12N9/22 , A61K38/00 , A61K48/00 , C07K2319/09 , C07K2319/21 , C07K2319/85 , C12N9/2462 , C12N15/102 , C12N15/113 , C12N15/115 , C12N15/63 , C12N2310/11 , C12N2310/16 , C12N2310/3519 , C12N2320/34 , C12N2800/80
摘要： The present invention provides RNA-guided gene editing systems and methods of use thereof.
摘要翻译：本发明提供了RNA引导的基因编辑系统及其使用方法。

2. 发明申请

WO2011051466A1 ANTI-IDIOTYPIC FIBRONECTIN-BASED BINDING MOLECULES AND USES THEREOF 审中-公开
标题翻译：基于抗体基于FIBRONECTIN的结合分子及其用途
公开(公告)号：WO2011051466A1
公开(公告)日：2011-05-05
申请号：PCT/EP2010/066503
申请日：2010-10-29
申请人： NOVARTIS AG , LOEW, Andreas , SAGOO, Jasbir
发明人： LOEW, Andreas , SAGOO, Jasbir
IPC分类号： C07K14/78 , C07K16/42
CPC分类号： C07K14/78 , C07K16/1018 , C07K2317/92 , C07K2318/20
摘要： The invention provides anti-idiotypic fibronectin type III (Fn3) polypeptides that bind to an antibody of a target antigen using an antigen mimic sequence present in one or more loop regions. The anti-idiotypic Fn3 polypeptides of the invention can be used to generate or bind half-life extender molecules such as Human Serum Albumin (HSA), for improved half life and stability. The invention also provides methods for generating, screening and using the anti-idiotypic Fn3 polypeptides in a variety of therapeutic and diagnostic applications.
摘要翻译：本发明提供使用存在于一个或多个环区中的抗原模拟序列结合靶抗原的抗体的抗独特型纤连蛋白III型（Fn3）多肽。本发明的抗独特型Fn3多肽可用于产生或结合半衰期延长分子例如人血清白蛋白（HSA），以改善半衰期和稳定性。本发明还提供了在各种治疗和诊断应用中产生，筛选和使用抗独特型Fn3多肽的方法。

3. 发明申请

WO2010069913A1 YEAST DISPLAY SYSTEMS 审中-公开
标题翻译： YEAST显示系统
公开(公告)号：WO2010069913A1
公开(公告)日：2010-06-24
申请号：PCT/EP2009/067066
申请日：2009-12-14
申请人： NOVARTIS AG , LOEW, Andreas
发明人： LOEW, Andreas
IPC分类号： C12N15/10 , C12N15/81 , C40B40/08
CPC分类号： C12N15/1037 , C07K14/395 , C07K14/78 , C07K2319/035 , C07K2319/70 , C07K2319/912 , C12N15/81 , C12N15/815 , C40B30/04 , C40B40/08 , C40B50/06
摘要： The present invention relates to the field of protein display libraries and library screening, In preferred embodiments, the present invention provides a three component system for display comprising a cell surface molecule, an adapter molecule and a display molecule.
摘要翻译：本发明涉及蛋白质显示库和文库筛选领域。在优选的实施方案中，本发明提供一种用于显示的三组分体系，其包括细胞表面分子，衔接子分子和显示分子。

4. 发明申请

WO2016028896A1 ANTI-CD123 CHIMERIC ANTIGEN RECEPTOR (CAR) FOR USE IN CANCER TREATMENT 审中-公开
标题翻译：抗CD123抗癌药物（CAR）用于癌症治疗
公开(公告)号：WO2016028896A1
公开(公告)日：2016-02-25
申请号：PCT/US2015/045898
申请日：2015-08-19
申请人： NOVARTIS AG , THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA , BROGDON, Jennifer , GILL, Saar , GLASS, David , KENDERIAN, Saad , LOEW, Andreas , MANNICK, Joan , MILONE, Michael , MURPHY, Leon , PORTER, David, L. , RUELLA, Marco , WANG, Yongqiang , WU, Qilong , ZHANG, Jiquan
发明人： BROGDON, Jennifer , GILL, Saar , GLASS, David , KENDERIAN, Saad , LOEW, Andreas , MANNICK, Joan , MILONE, Michael , MURPHY, Leon , PORTER, David, L. , RUELLA, Marco , WANG, Yongqiang , WU, Qilong , ZHANG, Jiquan
IPC分类号： C07K16/2866 , C07K16/28 , A61K39/395
CPC分类号： C07K16/2866 , A61K39/0011 , A61K2039/505 , A61K2039/507 , A61K2039/5156 , C07K16/244 , C07K2317/24 , C07K2317/622 , C07K2317/73 , C07K2317/76 , C07K2319/03 , C07K2319/33 , C12N5/0636
摘要： The invention provides compositions and methods for treating diseases associated with expression of CD 123. The invention also relates to chimeric antigen receptor (CAR) specific to CD123, vectors encoding the same, and recombinant cells comprising the CD123 CAR. The invention also includes methods of administering a genetically modified cell expressing a CAR that comprises a CD123 binding domain.
摘要翻译：本发明提供用于治疗与CD123表达相关的疾病的组合物和方法。本发明还涉及CD123特异性的嵌合抗原受体（CAR），编码它的载体，以及包含CD123 CAR的重组细胞。本发明还包括施用表达包含CD123结合结构域的CAR的遗传修饰的细胞的方法。

5. 发明申请

WO2016014553A1 SORTASE SYNTHESIZED CHIMERIC ANTIGEN RECEPTORS 审中-公开
标题翻译：筛选出合成的抗原基因受体
公开(公告)号：WO2016014553A1
公开(公告)日：2016-01-28
申请号：PCT/US2015/041363
申请日：2015-07-21
申请人： NOVARTIS AG , THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA , BROGDON, Jennifer , GUIMARAES, Carla , HASTEWELL, John , LOEW, Andreas
发明人： BROGDON, Jennifer , GUIMARAES, Carla , HASTEWELL, John , LOEW, Andreas
IPC分类号： C07K19/00 , C12N15/52 , C12N15/62 , C12N15/63 , C12N15/87 , C12N9/16 , C12N9/52
CPC分类号： C12N9/52 , C07K16/00 , C07K2319/00 , C07K2319/02 , C07K2319/03 , C07K2319/30 , C07K2319/70
摘要： Sortase molecules and methods described herein allow for the construction of a CAR or CAR member, e.g., in situ , on a CARX, e.g., CART, cell. For example, sortase mediated transfer of an antigen binding domain, e.g., a scFv, onto a CAR member having a sortase acceptor motif in place of an antigen binding domain can provide for a complete CAR member on a cell wherein the cell does not comprise nucleic acid that encodes the complete CAR member.
摘要翻译：本文所述的分选酶分子和方法允许在CARX例如CART细胞上构建CAR或CAR成员，例如原位构建。例如，分离酶介导的抗原结合结构域例如scFv转移到具有分解酶受体基序的CAR成员代替抗原结合结构域可以在细胞上提供完整的CAR成员，其中细胞不包含核酸编码完整CAR成员的酸。

6. 发明申请

WO2015142675A2 TREATMENT OF CANCER USING CHIMERIC ANTIGEN RECEPTOR 审中-公开
标题翻译：用嵌合型抗原受体治疗癌症
公开(公告)号：WO2015142675A2
公开(公告)日：2015-09-24
申请号：PCT/US2015/020606
申请日：2015-03-13
申请人： NOVARTIS AG , THE TRUSTEES OF UNIVERSITY OF PENNSYLVANIA , LOEW, Andreas , MILONE, Michael, C. , POWELL, Daniel, J. , ZHAO, Yangbing
发明人： LOEW, Andreas , MILONE, Michael, C. , POWELL, Daniel, J. , ZHAO, Yangbing
IPC分类号： A61K39/00
CPC分类号： C12N5/0638 , A61K39/0011 , A61K39/39 , A61K39/3955 , A61K2039/505 , A61K2039/5156 , A61K2039/55527 , C07K16/28 , C07K16/2863 , C07K16/30 , C07K16/32 , C07K2317/22 , C07K2317/569 , C07K2317/622 , C07K2317/77 , C07K2317/92 , C07K2319/00 , C07K2319/03 , C07K2319/32 , C12N5/0636 , C12N2510/00 , A61K2300/00
摘要： The invention provides compositions and methods for treating diseases associated with expression of a cancer associated antigen as described herein. The invention also relates to chimeric antigen receptor (CAR) specific to a cancer associated antigen as described herein, vectors encoding the same, and recombinant T cells comprising the CARs of the present invention. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises an antigen binding domain that binds to a cancer associated antigen as described herein.
摘要翻译：本发明提供了用于治疗与本文所述癌症相关抗原表达相关的疾病的组合物和方法。本发明还涉及对如本文所述的癌症相关抗原特异性的嵌合抗原受体（CAR），编码其的载体，以及包含本发明的CAR的重组T细胞。本发明还包括施用表达CAR的遗传修饰的T细胞的方法，所述CAR包含如本文所述的与癌症相关抗原结合的抗原结合结构域。

7. 发明申请

WO2013067355A1 LOW DENSITY LIPOPROTEIN-RELATED PROTEIN 6 (LRP6) - HALF LIFE EXTENDER CONSTRUCTS 审中-公开
标题翻译：低密度脂蛋白相关蛋白6（LRP6） - 半衰期延长结构
公开(公告)号：WO2013067355A1
公开(公告)日：2013-05-10
申请号：PCT/US2012/063330
申请日：2012-11-02
申请人： NOVARTIS AG , JENKINS, David , LEI, Ming , LOEW, Andreas , ZHOU, Li
发明人： JENKINS, David , LEI, Ming , LOEW, Andreas , ZHOU, Li
IPC分类号： C07K16/28
CPC分类号： A61K47/48415 , A61K38/385 , A61K39/39558 , A61K47/6811 , A61K2039/505 , C07K14/76 , C07K14/765 , C07K14/775 , C07K16/28 , C07K16/30 , C07K16/46 , C07K2317/35 , C07K2317/55 , C07K2317/622 , C07K2317/76 , C07K2317/94 , C07K2319/30 , C07K2319/31 , C12N2740/15041 , Y10S424/809
摘要： The present invention relates to LRP6 constructs that bind to LRP6 receptor. The LRP6 constructs comprise at least one LRP6 binding moiety and a half-life extender molecule such that the LRP6 construct inhibit the Wnt signaling pathway without potentiation of the Wnt signal. The LRP6 constructs also have an increased half-life to provide more time for the therapeutic benefit.
摘要翻译：本发明涉及结合LRP6受体的LRP6构建体。 LRP6构建体包含至少一个LRP6结合部分和半衰期延长分子，使得LRP6构建体在不增强Wnt信号的情况下抑制Wnt信号传导途径。 LRP6构建体还具有增加的半衰期以提供更多的治疗益处的时间。

8. 发明申请

WO2012066075A1 METHODS OF TREATING FGF21-ASSOCIATED DISORDERS 审中-公开
标题翻译：治疗FGF21相关疾病的方法
公开(公告)号：WO2012066075A1
公开(公告)日：2012-05-24
申请号：PCT/EP2011/070344
申请日：2011-11-17
申请人： NOVARTIS AG , IRM LLC , BOETTCHER, Brian R. , CAPLAN, Shari L. , DANIELS, Douglas S. , GEIERSTANGER, Bernhard H. , HAMAMATSU, Norio , LICHT, Stuart , LOEW, Andreas , WELDON, Stephen Craig
发明人： BOETTCHER, Brian R. , CAPLAN, Shari L. , DANIELS, Douglas S. , GEIERSTANGER, Bernhard H. , HAMAMATSU, Norio , LICHT, Stuart , LOEW, Andreas , WELDON, Stephen Craig
IPC分类号： C07K14/50
CPC分类号： A61K38/1825 , A61K47/593 , A61K47/60 , C07K14/50 , C07K2319/00 , C07K2319/30 , C07K2319/31
摘要： The invention relates to the identification of new polypeptide and protein variants of fibroblast growth factor 21 (FGF21) that have improved pharmaceutical properties. Also disclosed are methods for treating FGF21-associated disorders, including metabolic conditions.
摘要翻译：本发明涉及具有改善的药物性质的成纤维细胞生长因子21（FGF21）的新多肽和蛋白质变体的鉴定。还公开了治疗FGF21相关疾病（包括代谢病症）的方法。

9. 发明申请

WO2016098078A2 DIMERIZATION SWITCHES AND USES THEREOF 审中-公开
标题翻译：二极管开关及其用途
公开(公告)号：WO2016098078A2
公开(公告)日：2016-06-23
申请号：PCT/IB2015/059796
申请日：2015-12-18
申请人： NOVARTIS AG , LOEW, Andreas , VASH, Brian
发明人： LOEW, Andreas , VASH, Brian
IPC分类号： C12N15/62
CPC分类号： C12N9/90 , C07K2319/09 , C07K2319/70 , C07K2319/81 , C12N9/12 , C12Y207/11001 , C12Y502/01008
摘要： The present invention provides gene editing systems comprising gene editing dimerization switches comprising a first and second gene editing switch domain that allow for the regulation of a gene editing function by the introduction, e.g., administration, of a gene editing dimerization molecule having the ability to bring together a first gene editing switch domain and a second gene editing switch domain. A regulated gene editing function provides, e.g., less off-target side effects, and increases the therapeutic window. The present invention also provides improved FKBP/FRB-based dimerization switches wherein the FRB switch domain or the FKBP switch domain, or both the FRB and FKBP switch domains, comprise one or more mutations that optimize performance, e.g., that alter, e.g., enhance the formation of a complex between the first switch domain, the second switch domain, and the dimerization molecule, rapamycin, or a rapalog, e.g., RAD001.
摘要翻译：本发明提供了基因编辑系统，其包括基因编辑二聚转换开关，其包括第一和第二基因编辑开关结构域，其允许通过引入（例如）施用具有带来能力的基因编辑二聚化分子来调节基因编辑功能一起是第一个基因编辑开关域和第二个基因编辑开关域。调节基因编辑功能提供例如较少的目标外副作用，并增加治疗窗口。本发明还提供改进的基于FKBP / FRB的二聚开关，其中FRB开关域或FKBP开关域，或FRB和FKBP开关结构域两者都包含优化性能的一个或多个突变，例如改变，例如增强在第一开关结构域，第二开关结构域和二聚化分子雷帕霉素或rapalog（例如RAD001）之间形成复合物。

10. 发明申请

WO2011051327A2 SMALL ANTIBODY-LIKE SINGLE CHAIN PROTEINS 审中-公开
标题翻译：小抗体样单链蛋白
公开(公告)号：WO2011051327A2
公开(公告)日：2011-05-05
申请号：PCT/EP2010/066243
申请日：2010-10-27
申请人： NOVARTIS AG , LOEW, Andreas
发明人： LOEW, Andreas
IPC分类号： C07K16/00
CPC分类号： C07K16/468 , C07K16/00 , C07K16/081 , C07K16/1232 , C07K16/14 , C07K16/16 , C07K16/464 , C07K2317/22 , C07K2317/569
摘要： The present invention provides a "ASCP-body" that comprises a bottomside epitope binding domain ("BEBD") comprising four Bottomside Binding Regions (BBR1, BBR2, BBR3, and BBR4). This epitope binding domain is in addition to the topside epitope binding domain (TEBD) formed by the three Complementarity Determining Regions (CDR1, CDR2, and CDR3). The invention encompasses mono-specific, bi-specific and multi-specific, and mono-valent, bi-valent and multi-valent ASCP-bodies. The invention also encompasses fusion proteins in which one or more ASCP-bodies with a BEBD is conjugated to one or more ASCP-bodies with a BEBD and, optionally, a TEBD; to one or more ASCP- bodies with a TEBD and, optionally, a BEBD; or to one or more non-ASCP-body entity(ies). The various ASCP-bodies in the bi-specific and multi-specific ASCP-bodies and fusion proteins may all bind to the same epitopes or antigens or may bind to different epitopes or antigens.
摘要翻译：本发明提供了包含四个底部结合区（BBR1，BBR2，BBR3和BBR4）的底部表位结合结构域（“BEBD”）的“ASCP体”。该表位结合结构域除了由三个互补决定区（CDR1，CDR2和CDR3）形成的顶侧表位结合结构域（TEBD）之外。本发明包括单特异性，双特异性和多特异性以及一价，一价和多价的ASCP体。本发明还包括融合蛋白，其中具有BEBD的一个或多个ASCP-体与一种或多种具有BEBD和任选的TEBD的ASCP-体共轭; 具有TEBD和可选的BEBD的一个或多个ASCP体; 或一个或多个非ASCP身体实体。双特异性和多特异性ASCP体和融合蛋白中的各种ASCP-体都可以结合相同的表位或抗原，或者可以结合不同的表位或抗原。

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