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1. 发明公开

EP2796150A1 NOVEL OLIGONUCLEOTIDE CONJUGATES AND USE THEREOF 有权
标题翻译：维也纳戴维顿的神经元寡妇
公开(公告)号：EP2796150A1
公开(公告)日：2014-10-29
申请号：EP12857568.5
申请日：2012-12-14
申请人： Bioneer Corporation
发明人： CHAE, Jeiwook , HAN, Boram , KIM, Han-na , PARK, Han Oh , YOON, Pyoung Oh , KIM, Sun Gi , JUNG, Kwang-Ju , KWON, Taewoo , CHOI, Jong Deok , LEE, Sam Young , JUNG, Eun-Jung
IPC分类号： A61K47/48 , C12N15/11 , A61K9/16 , A61K48/00
CPC分类号： A61K47/42 , A61K31/713 , A61K47/549 , A61K47/551 , A61K47/60 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2310/3513 , C12N2310/3515 , C12N2320/32
摘要： The present invention provides a double-stranded RNA structure, which comprises a polymer compound covalently bonded to a double-helix oligo RNA useful for the treatment of diseases, particularly cancer, in order to enhance the delivery of the double-helix oligo RNA, and further comprises a target-specific ligand bonded thereto, a preparation method thereof, and a technique of delivering the double-helix oligo RNA in a target-specific manner using the RNA structure. A nanoparticle composed of the ligand-bonded double-helix oligo RNA structures can efficiently deliver the double-helix oligo RNA to a target, and thus can exhibit the activity of the double-helix oligo RNA even when the double-helix oligo RNA is administered at a relatively low concentration. Also, it can prevent the non-specific delivery of the double-helix oligo RNA into other organs and cells. Accordingly, the ligand-bonded double-stranded RNA structure can be used for the treatment for various diseases, particularly cancer, and can also be effectively used as a new type of double-helix oligo RNA delivery system. Particularly, the ligand-bonded double-stranded RNA structure can be effectively used for the treatment of diseases, including cancer and infectious diseases. Moreover, the present invention relates to a hybrid conjugate, which comprises a hydrophilic material and hydrophobic material bonded to both ends of an antisense oligonucleotide (ASO) by a covalent bond in order to enhance the in vivo stability of the ASO, a method for preparing the hybrid conjugate, and a nanoparticle composed of the conjugates. The ASO-polymer conjugate according to the invention can increase the in vivo stability of the ASO, making it possible to efficiently deliver the therapeutic ASO into cells. Also, the ASO-polymer conjugate can exhibit the activity of the ASO even when it is administered at a relatively low concentration.
摘要翻译：本发明提供双链RNA结构，其包含与用于治疗疾病，特别是癌症的双螺旋寡聚RNA共价键合的高分子化合物，以增强双螺旋寡聚RNA的递送，以及还包括与其键合的靶特异性配体，其制备方法，以及使用RNA结构以目标特异性方式递送双螺旋寡聚RNA的技术。由配体键合的双螺旋寡聚RNA结构组成的纳米粒子可以有效地将双螺旋寡聚RNA递送到靶，因此即使施用双螺旋寡核苷酸RNA也能显示双螺旋寡聚RNA的活性浓度相对较低。此外，它可以防止双螺旋寡聚RNA非特异性递送到其他器官和细胞中。因此，配体键合的双链RNA结构可以用于各种疾病，特别是癌症的治疗，也可以有效地用作新型双螺旋寡聚RNA递送系统。特别地，配体键合的双链RNA结构可以有效地用于治疗包括癌症和感染性疾病在内的疾病。此外，本发明涉及一种杂合缀合物，其包含通过共价键与反义寡核苷酸（ASO）的两端键合的亲水性材料和疏水性材料，以增强ASO的体内稳定性，制备方法杂交缀合物和由缀合物组成的纳米颗粒。根据本发明的ASO-聚合物缀合物可以增加ASO的体内稳定性，使得可以有效地将治疗性ASO递送到细胞中。此外，即使以相对低的浓度施用，ASO-聚合物缀合物也可以表现出ASO的活性。

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