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公开(公告)号：US20210228608A1
公开(公告)日：2021-07-29
申请号：US17226050
申请日：2021-04-08
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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公开(公告)号：US20190224226A9
公开(公告)日：2019-07-25
申请号：US15914045
申请日：2018-03-07
IPC分类号： A61K31/7036 , B65D79/00 , A61K45/06
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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公开(公告)号：US20160074425A1
公开(公告)日：2016-03-17
申请号：US14866960
申请日：2015-09-27
IPC分类号： A61K31/7036
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
摘要翻译： 提供了一类新的具有5“位烷基的假三糖氨基糖苷类，显示出有效的终止密码子突变读取活性，低细胞毒性和对真核翻译系统的高选择性。 还提供了含有该组合物的药物组合物及其在治疗遗传疾病中的用途，以及制备这些氨基糖苷类的方法。 所公开的氨基糖苷类可以由通式I表示：或其药学上可接受的盐，其中R 1选自烷基，环烷基和芳基; 并且所有其他变量和特征如说明书中所述。
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公开(公告)号：US20200078386A1
公开(公告)日：2020-03-12
申请号：US16550121
申请日：2019-08-23
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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公开(公告)号：US20180200276A1
公开(公告)日：2018-07-19
申请号：US15914045
申请日：2018-03-07
IPC分类号： A61K31/7036 , B65D79/00 , A61K45/06
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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公开(公告)号：US20170182078A1
公开(公告)日：2017-06-29
申请号：US15453990
申请日：2017-03-09
IPC分类号： A61K31/7036 , B65D79/00 , A61K45/06
摘要： A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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