发明名称 Compound having read-through activity
摘要 [Problem] Provision of a novel compound having read-through activity and a drug for the treatment of nonsense mutation-type disease containing this compound.;[Solution] A compound represented by the following general formula (1):; and a pharmaceutical composition containing this compound.
申请公布号 US9371274(B2) 申请公布日期 2016.06.21
申请号 US201414291861 申请日期 2014.05.30
申请人 THE UNIVERSITY OF TOKYO 发明人 Hayashi Yoshio;Taguchi Akihiro;Yakushiji Fumika;Yamazaki Yuri;Matsuda Ryoichi;Shiozuka Masataka
分类号 A61K31/16;A61K31/197;C07C243/12;C07C243/34 主分类号 A61K31/16
代理机构 Troutman Sanders LLP 代理人 Sullivan Clark G.;Troutman Sanders LLP
主权项 1. A compound represented by formula (1):in which R1 and R2 are each independently hydrogen, an optionally substituted C1-C6 alkyl group, C2-C6 alkenyl group, C2-C6 alkynyl group, C6-C10 aralkyl group, C6-C10 aralkenyl group, sulfonyl group, cyclic amine, or guanidyl group, R1 and R2 also may form, together with the nitrogen atom to which they are bonded, a five-membered or six-membered heterocycle or heteroaryl bonded via N, may also contain 1-3 additional hetero atoms selected from the group consisting of O, N, and S as ring constituent members, and the heterocycle or heteroaryl may be substituted by a C1-C6 alkyl group, C2-C6 alkenyl, or C2-C6 alkynyl, C6-C10 aralkyl group, or C6-C10 aralkenyl group, one or both of R1 and R2 may be an amino acid residue introduced via an amide bond formed together with the nitrogen atom to which they are bonded; X is N(R3)(R4), an N-linked amino acid residue, or a C1-C6 alkyl group containing an amino group having a substituent, where the substituent of the amino group is hydrogen, an optionally substituted C1-C6 alkyl group, C2-C6 alkenyl group, C2-C6 alkynyl group, C6-C10 aralkyl group, C6-C10 aralkenyl group, cyclic amine, or guanidyl group, where R3 and R4 are each independently hydrogen or an optionally substituted C1-C6 alkyl group, R3 and R4 also may form, together with the nitrogen atom to which they are bonded, a five-membered or six-membered heterocycle or heteroaryl bonded via N, may also contain 1-3 additional hetero atoms selected from the group consisting of O, N, and S as ring constituent members, where the heterocycle or heteroaryl may be substituted by a C1-C6 alkyl group, C2-C6 alkenyl group, or C2-C6 alkynyl group, C6-C10 aralkyl group, C6-C10 aralkenyl group; R5 is a hydrogen, C1-C4 alkyl group, C2-C4 alkenyl group, or C2-C4 alkynyl group; R6 is a C1-C4 alkyl group, C2-C4 alkenyl group, C2-C4 alkynyl group, C3-C6 cycloalkyl group, carboxyl group, carboxylate group, alkyl group having a carboxyl group or carboxylate group; C2-C6 alkenyl group, C2-C6 alkynyl group, C6-C10 aralkyl group, C6-C10 aralkenyl group, cyclic amine, or guanidyl group, R5 and R6 also may form, together with the nitrogen atom to which they are bonded, a five-membered or six-membered heterocycle or heteroaryl bonded via N, may also contain 1-3 additional hetero atoms selected from the group consisting of O, N, and S as ring constituent members, where the heterocycle or heteroaryl may be substituted by a C1-C6 alkyl group, C2-C6 alkenyl group, or C2-C6 alkynyl group, C6-C10 aralkyl group, C6-C10 aralkenyl group; R7 is a C1-C6 alkyl group, substituted or unsubstituted aralkyl group, or C3-C7 cycloalkyl group, where the substituent of the substituted aralkyl group is a halogen, C1-C4 alkyl group, alkoxy group, hydroxy group, nitro group, amino group, C1-C6 acyl group, or amino group modified by an alkyl group, or a sulfonic acid group; and n is an integer of 0-3,or a pharmaceutically acceptable salt or solvate of the compound.
地址 Tokyo JP