[EN] M6PR CELL SURFACE RECEPTOR BINDING COMPOUNDS AND CONJUGATES<br/>[FR] COMPOSÉS ET CONJUGUÉS DE LIAISON AU RÉCEPTEUR DE SURFACE CELLULAIRE M6PR
申请人:LYCIA THERAPEUTICS INC
公开号:WO2023288015A1
公开(公告)日:2023-01-19
The present disclosure provides a class of compounds including a ligand moiety that specifically binds to a cell surface mannose-6-phosphate receptor (M6PR). The M6PR binding compounds can trigger the receptor to internalize into the cell a bound compound. The ligand moieties of this disclosure can be linked to a variety of moieties of interest without impacting the specific binding to, and function of, the M6PR. Also provided are compound that are conjugates of the ligand moieties linked to a biomolecule, such as an antibody, which conjugates can harness cellular pathways to remove specific target proteins from the cell surface or the extracellular milieu. For example, the conjugates described herein may sequester and/or degrade a target molecule of interest in a cell's lysosome. Also provided are methods of using the conjugates to target a protein for sequestration and/or lysosomal degradation.
本公开提供了一类化合物,包括一个配体基团,该基团专门结合细胞表面的甘露醇-6-磷酸受体(M6PR)。M6PR结合化合物可以触发受体内部化到细胞中结合的化合物。本公开的配体基团可以与各种感兴趣的基团链接,而不会影响对M6PR的特异性结合和功能。还提供了化合物,它们是与生物分子(例如抗体)连接的配体基团的共轭物,这些共轭物可以利用细胞途径从细胞表面或细胞外环境中去除特定的靶蛋白质。例如,本文所述的共轭物可以在细胞溶酶体中固定和/或降解感兴趣的靶分子。还提供了使用共轭物来靶向蛋白质进行固定和/或溶酶体降解的方法。