基本信息:
- 专利标题： COMPOUNDS FOR TREATING MITOCHONDRIAL DNA DISORDERS
- 申请号：PCT/GB2022/052910 申请日：2022-11-16
- 公开(公告)号：WO2023089313A1 公开(公告)日：2023-05-25
- 发明人： SPINAZZOLA, Antonella , HOLT, Ian , PANTIC, Boris , IVES, Daniel
- 申请人： UCL BUSINESS LTD , UNITED KINGDOM RESEARCH AND INNOVATION
- 申请人地址： University College London; Polaris House
- 专利权人： UCL BUSINESS LTD,UNITED KINGDOM RESEARCH AND INNOVATION
- 当前专利权人： UCL BUSINESS LTD,UNITED KINGDOM RESEARCH AND INNOVATION
- 当前专利权人地址： University College London; Polaris House
- 代理机构： WITHERS & ROGERS LLP
- 优先权： GB2116499.1 2021-11-16
- 主分类号： A61K38/46
- IPC分类号： A61K38/46 ; A61K31/7004 ; A61K31/19 ; A61P25/28 ; A61P21/00 ; A61P43/00
摘要：
There is described a compound for use in the treatment of a mitochondrial DNA disorder, wherein the compound is a glycolysis inhibitor, an inhibitor of glutamine consumption or is L-asparaginase or pegaspargase. The glycolysis inhibitor may be a glucose analogue such as 2-deoxy-D-glucose (2DG) or 5-thioglucose (5TG). Also described is a method of treating a mitochondrial DNA disorder comprising administering a therapeutically effective amount of the compound above to a patient suffering from a mitochondrial DNA disorder. In addition, there is described a method of reducing the mtDNA heteroplasmy in the cells of a patient comprising administering a therapeutically effective amount of the compound above to the patient.
IPC结构图谱:
| A | 人类生活必需 |
| --A61 | 医学或兽医学；卫生学 |
| ----A61K | 医用、牙科用或梳妆用的配制品 |
| ------A61K38/00 | 含肽的医药配制品 |
| --------A61K38/04 | .在完全确定的序列中最多有20个氨基酸的肽；其衍生物 |
| ----------A61K38/17 | ..来源于动物；来源于人类 |
| ------------A61K38/46 | ...水解酶（3） |