发明名称 Premature-termination-codons readthrough compounds
摘要 Premature termination codons readthrough compounds, composition thereof, and methods of making and using the same are provided.
申请公布号 US9598395(B2) 申请公布日期 2017.03.21
申请号 US201314387212 申请日期 2013.03.15
申请人 THE REGENTS OF THE UNIVERSITY OF CALIFORNIA 发明人 Gatti Richard A.;Du Liutao;Hu Hailiang;Damoiseaux Robert;Jung Michael E.;Ku Jin-Mo;Completo Gladys
分类号 C07D401/04;C07D401/14 主分类号 C07D401/04
代理机构 Foley Hoag LLP 代理人 Foley Hoag LLP ;Halstead David P.
主权项 1. A compound of formula Ia: where R1a is 2-hydroxylphenyl, 2-alkoxyphenyl, 3-hydroxylphenyl, or 3-alkoxyphenyl; X1 is S, O, NH, or N(C1-3-alkyl); R2a is (CH2)m; m is 1, 2, or 3; Y1 is O, S, or NH; R3a is hydroxy or —NR7R7a; R7 is hydrogen or C1-3-alkyl; R7a is hydroxyalkyl; phenyl substituted with 1, 2, or 3 R8 groups; phenyl substituted with two independently selected halo; each R8 is independently hydroxy or haloalkyl; and each R9, when present, is independently hydroxy, alkoxy, halo, haloalkyl, or C1-6-alkyl; and provided that 1) when X1 is S, R2a is —CH2—, Y1 is O, and R3a is —NR7R7a and R7 is hydrogen, then R7a is not 2-methoxyphenyl; and 2) when R1a is 2-methoxyphenyl, X1 is S, R2a is —CH2—, Y1 is O, and R3a is —NR7R7a and R7 is hydrogen, then R7a is not 4-methoxyphenyl; or a pharmaceutically acceptable salt thereof.
地址 Oakland CA US