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    • 5. 发明申请
    • SMALL ANTIBODY-LIKE SINGLE CHAIN PROTEINS
    • 小型抗体样单链蛋白
    • WO2011051327A3
    • 2011-06-30
    • PCT/EP2010066243
    • 2010-10-27
    • NOVARTIS AGLOEW ANDREAS
    • LOEW ANDREAS
    • C07K16/00
    • C07K16/468C07K16/00C07K16/081C07K16/1232C07K16/14C07K16/16C07K16/464C07K2317/22C07K2317/569
    • 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.
    • 本发明提供了包含含有四个底部结合区(BBR1,BBR2,BBR3和BBR4)的底侧表位结合结构域(“BEBD”)的“ASCP-体”。 该表位结合域除了由三个互补决定区(CDR1,CDR2和CDR3)形成的上游表位结合域(TEBD)之外。 本发明包括单特异性,双特异性和多特异性以及单价,二价和多价ASCP体。 本发明还包括融合蛋白,其中一个或多个具有BEBD的ASCP-体与BEBD和任选的TEBD缀合至一个或多个ASCP-体; 到一个或多个具有TEBD和可选的BEBD的ASCP体; 或一个或多个非ASCP机构实体。 双特异性和多特异性ASCP体和融合蛋白中的各种ASCP体可全部结合相同的表位或抗原或可结合不同的表位或抗原。
    • 6. 发明申请
    • DIMERIZATION SWITCHES AND USES THEREOF
    • 二化化开关及其用途
    • WO2016098078A3
    • 2016-10-27
    • PCT/IB2015059796
    • 2015-12-18
    • NOVARTIS AGLOEW ANDREASVASH BRIAN
    • LOEW ANDREASVASH BRIAN
    • C12N15/62C12N9/12C12N9/90
    • C12N9/90C07K2319/09C07K2319/70C07K2319/81C12N9/12C12Y207/11001C12Y502/01008
    • 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.
    • 本发明提供了基因编辑系统,其包含基因编辑二聚化开关,所述基因编辑二聚化开关包含第一和第二基因编辑开关结构域,所述第一和第二基因编辑开关结构域允许通过引入(例如施用)编码二聚化分子的基因来调节基因编辑功能, 一起构成第一基因编辑开关域和第二基因编辑开关域。 受管理的基因编辑功能提供了例如更少的脱靶副作用,并增加了治疗窗口。 本发明还提供了改进的基于FKBP / FRB的二聚化开关,其中FRB开关结构域或FKBP开关结构域或FRB和FKBP开关结构域两者包含一个或多个优化性能的突变,例如改变例如增强 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雷帕霉素例如RAD001之间形成复合物。