发明名称 SIALIC ACID ANALOGS
摘要 The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
申请公布号 US2015038693(A1) 申请公布日期 2015.02.05
申请号 US201414461295 申请日期 2014.08.15
申请人 ULTRAGENYX PHARMACEUTICAL INC. 发明人 KAKKIS Emil;JUNGLES Steven;ZHAO He
分类号 C07H7/02;C07D493/08 主分类号 C07H7/02
代理机构 代理人
主权项 1. A compound having structural Formula (I):or a pharmaceutically acceptable salt or solvate thereof; wherein: R2, R3, R4, and R6 are independently hydrogen or a moiety selected from structural formula (a), (b), (c), (d), (e), (f), and (g): R1 is a moiety selected from structural formula (a), (b), (c), (f), and (g); or a nucleoside phosphate moiety; X is oxygen or sulfur; L1 and L2 are each independently a covalent bond, —O—, or —NR10a—; R10a is hydrogen or optionally substituted alkyl; R1a and R2a are each independently hydrogen, optionally substituted alkyl, optionally substituted heteroalkyl, optionally substituted aryl, optionally substituted heteroaryl, optionally substituted carbocyclyl, optionally substituted heterocyclyl, —Xa—C(O)—O—R11a, or —Xa—O—C(O)—O—R11a; Xa is optionally substituted alkylene; each R11a is independently hydrogen, optionally substituted alkyl, or optionally substituted heteroalkyl; R3a is optionally substituted alkyl; or alternatively, R3a, together with the carboxyl moiety to which it is attached, form a monopeptidyl or dipeptidyl group; each R8a and R9a is independently hydrogen or optionally substituted alkyl; m is 1 or 2; Z is hydrogen, lower alkyl, an amide group, a lactam group, an ester group, a lactone group, an urea group, a cyclic urea group, a carbonate group, a cyclic carbonate group, a carbamate group, a cyclic carbamate group, or a moiety selected from (a), (b), (c), and (f); R5 is hydrogen, G+, optionally substituted alkyl, or a moiety selected from (a), (b), (c), (f), and (g); and G+ is a charged organic amine moiety; or alternatively, OR3 and OR6 are taken together to form a lactone structure represented by Formula (Ia):and with the following provisos: (a) at least one of R1, R2, R3, R4, R5, and R6 is not H; and (b) R3a is not optionally substituted alkyl unless (1) OR3 and OR6 are taken together to form a lactone structure of Formula (Ia), or (2) R5 is (f).
地址 Novato CA US