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    • 103. 发明申请
    • TARGETED THERAPEUTICS
    • 目标治疗
    • WO2014004465A1
    • 2014-01-03
    • PCT/US2013/047550
    • 2013-06-25
    • THE BRIGHAM AND WOMEN'S HOSPITAL, INC.
    • LEE, Richard, T.PATWARI, ParthLOFFREDO, FrancescoPANCOAST, James
    • C07K19/00C07K14/00C12N15/63C12N5/10A61K38/16A61P19/00A61P25/00A61P27/00
    • C07K14/65A61K38/00A61K38/16A61K38/29A61K38/30C07K14/47C07K14/635C07K2319/00C07K2319/01C07K2319/33
    • The present invention relates to compositions, methods and kits for delivery of active agents (X) to cells or tissues that express proteoglycans. More specifically, the present invention relates to conjugates comprising (HB-linker) n -X m -(linker-HB) o , where HB is a heparin binding protein, X is an active agents such as a therapeutic protein (including a functional fragment or variant thereof), or a therapeutic small molecule, linker is a linker entity and m is an integer of at least 1, and n+o is an integer of at least 1. Other aspects relate to use of a HB-X conjugate in methods to deliver active agents to cartilage for the treatment of cartilage related disease and disorders, the use of the HB-X conjugate in methods to deliver active agents to neuronal tissues (e.g., brain and spinal cord) for the treatment of neurological disorders, and the use of the HB-X conjugates in methods for the treatment of eye conditions and disorders and inflammatory conditions and disorders.
    • 本发明涉及用于将活性剂(X)递送至表达蛋白聚糖的细胞或组织的组合物,方法和试剂盒。 更具体地说,本发明涉及包含(HB-接头)n-Xm-(接头-HB)o的缀合物,其中HB是肝素结合蛋白,X是活性剂,例如治疗性蛋白质(包括功能片段或 变体)或治疗性小分子,接头是连接体实体,m是至少为1的整数,n + o是至少为1的整数。其它方面涉及在方法中使用HB-X缀合物 将软骨中的活性剂递送到软骨相关疾病和病症的治疗中,在将神经元组织(例如脑和脊髓)递送活性剂用于治疗神经障碍的方法中使用HB-X缀合物,以及 在治疗眼部疾病和病症以及炎性疾病和病症的方法中使用HB-X缀合物。