Summit plc , 91 Milton Park, Abingdon, Oxfordshire, OX14 4RY, United Kingdom.
J Med Chem. 2011 May 12;54(9):3241-50. doi: 10.1021/jm200135z. Epub 2011 Apr 15.
A series of novel 2-arylbenzoxazoles that upregulate the production of utrophin in murine H2K cells, as assessed using a luciferase reporter linked assay, have been identified. This compound class appears to hold considerable promise as a potential treatment for Duchenne muscular dystrophy. Following the delineation of structure-activity relationships in the series, a number of potent upregulators were identified, and preliminary ADME evaluation is described. These studies have resulted in the identification of 1, a compound that has been progressed to clinical trials.
已经鉴定出一系列新型 2-芳基苯并恶唑，它们可通过与报告基因连接的检测方法在鼠 H2K 细胞中增加肌联蛋白的产生。此类化合物有望成为治疗杜氏肌营养不良症的一种潜在疗法。在对该系列化合物进行了构效关系的研究后，确定了一些有效的上调剂，并对初步的 ADME 评估进行了描述。这些研究的结果确定了化合物 1，它已进入临床试验阶段。
