公开(公告)号：WO2008121768A3

公开(公告)日：2009-12-30

申请号：PCT/US2008058576

申请日：2008-03-28

Applicant: UNIV TENNESSEE RES FOUNDATION ,  JABLONSKI MONICA M

Inventor： JABLONSKI MONICA M

IPC: A61K31/715

CPC classification number: A61K31/726

Abstract: Disclosed are novel methods of treatment for retinal diseases and conditions including age- related macular degeneration, genetic-based retinal degenerations and retinal detachment. A novel glycan binding protein thought to be a cell surface receptor has been discovered in the retina. The retinal glycan binding receptor is shown to play an important role in promoting assembly of outer segment (OS) membranes by the photoreceptor cells of the eye, a process that is essential for vision. Based on the finding that certain sugars can bind with very high affinity to the retinal glycan receptor and stimulate its function, the invention provides novel therapeutic agents for treatment of retinal diseases that are multivalent N-linked glycans. Preferred pharmaceutical compositions in accordance with the present invention comprise active agents having the general formula: (Gal-GlcNAc)n-Man3-GlcN Ac2, where n is 1-4. Particularly preferred multivalent glycans are galactosylated, biantennary (NA2), and asialo, galactosylated, triantennary (NA3) oligosaccharides.

Abstract translation: 公开了用于视网膜疾病和病症的新型治疗方法，包括年龄相关性黄斑变性，遗传性视网膜变性和视网膜脱离。 在视网膜中已经发现被认为是细胞表面受体的新型聚糖结合蛋白。 显示视网膜聚糖结合受体在促进眼睛光感受器细胞外壳（OS）膜的组装中发挥重要作用，这是对视力至关重要的过程。 基于某些糖可以以非常高的亲和力结合到视网膜聚糖受体并刺激其功能的发现，本发明提供用于治疗作为多价N-连接聚糖的视网膜疾病的新型治疗剂。 根据本发明的优选药物组合物包含具有以下通式的活性剂：（Gal-GlcNAc）n-Man3-GlcN Ac2，其中n为1-4。 特别优选的多价聚糖是半乳糖基化的，双向的（NA2）和脱唾液酸，半乳糖基化的三硝基（NA3）寡糖。