发明名称 |
COMPOSITIONS FOR PROMOTING READTHROUGH OF PREMATURE TERMINATION CODONS, AND METHODS OF USING THE SAME |
摘要 |
Disclosed are compounds of general formula (I) that promote readthrough of a premature termination codon (PTC) of an RNA molecule in a translation system, and their use, alone or in combination with other compounds, such as aminoglycoside, to treat diseases or disorders ameliorated by modulation of a premature termination codon (PTC) of an RNA molecule in a translation system. The disorder or disease may be Dystrophic epidermolysis bullosa, Batten disease, Duchenne muscular dystrophy, cancer, and spinal muscular atrophy. Ar-L-B (I) |
申请公布号 |
WO2017049409(A1) |
申请公布日期 |
2017.03.30 |
申请号 |
WO2016CA51119 |
申请日期 |
2016.09.23 |
申请人 |
THE CENTRE FOR DRUG RESEARCH AND DEVELOPMENT;THE UNIVERSITY OF BRITISH COLUMBIA |
发明人 |
ROBERGE, Michel;BARADARAN-HERAVI, Alireza;BALGI, Aruna D.;ZIMMERMANN, Carla D.;KRAUSE, Alexandra;PFEIFER, Thomas Arthur;JAQUITH, James Brian;TAN, Jason Samuel;SHIDMOOSSAVEE, Fahimeh S.;ARNS, Stephen Paul;BAUDELET, Davy Jérémy |
分类号 |
C07D209/48;A61K31/166;A61K31/18;A61K31/196;A61K31/343;A61K31/403;A61K31/4035;A61K31/404;A61K31/416;A61K31/4164;A61K31/4184;A61K31/423;A61K31/4704;A61K31/472;A61K31/496;A61K31/498;A61K31/517;A61K31/5375;A61K31/538;C07C237/30;C07C311/39;C07D207/408;C07D209/34;C07D209/46;C07D209/52;C07D209/54;C07D211/88;C07D215/12;C07D215/24;C07D215/26;C07D217/24;C07D231/56;C07D233/02;C07D233/72;C07D235/02;C07D239/91;C07D241/42;C07D263/58;C07D265/33;C07D265/36;C07D307/00;C07D401/06;C07D403/06;C07D405/06;C12N15/00;C12P21/00 |
主分类号 |
C07D209/48 |
代理机构 |
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代理人 |
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