申请人:The Brigham and Women's Hospital, Inc.
公开号:EP3369747A1
公开(公告)日:2018-09-05
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-Xm-(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 interger 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.
本发明涉及向表达蛋白多糖的细胞或组织递送活性剂(X)的组合物、方法和试剂盒。更具体地说,本发明涉及包括(HB-连接体)n-Xm-(连接体-HB)o 的共轭物,其中 HB 是肝素结合蛋白,X 是活性剂,如治疗性蛋白(包括其功能片段或变体)或治疗性小分子,连接体是连接体实体,m 是至少 1 的整数,n+o 是至少 1 的整数。其他方面涉及将 HB-X 共轭物用于向软骨输送活性剂的方法,以治疗软骨相关疾病和失调;将 HB-X 共轭物用于向神经元组织(如脑和脊髓)输送活性剂的方法,以治疗神经元相关疾病和失调、HB-X轭合物在治疗眼部疾病和炎症疾病的方法中的应用。