DANA ATIA-GLIKIN;TECHNION RESEARCH AND DEVELOPMENT FOUNDATION LIMITED;JEYAKUMAR KANDASAMY;TIMOR BAASOV;VALERY BELAKHOV
发明人:
DANA ATIA-GLIKIN,JEYAKUMAR KANDASAMY,VALERY BELAKHOV,TIMOR BAASOV
申请号：
IL26649519
公开号：
IL266495D0
申请日:
2019.05.07
申请国别(地区):
IL
年份:
2019
代理人:
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 are represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein R 1 is selected from the group consisting of alkyl, cycloalkyl and aryl； and all other variables and features are as described in the specification.
