发明名称 Retinal derivatives and methods for the use thereof for the treatment of visual disorders
摘要 Compositions of and methods for using synthetic retinal derivatives as retinoid replacements and opsin agonists are provided.
申请公布号 US9162978(B2) 申请公布日期 2015.10.20
申请号 US201213619631 申请日期 2012.09.14
申请人 University of Washington 发明人 Palczewski Krzysztof;Batten Matthew
分类号 A61K31/22;A61K31/225;C07C403/14;C07C403/10;C07C403/12;C07C403/20;A61K31/215 主分类号 A61K31/22
代理机构 McDermott Will & Emery LLP 代理人 Mahoney Jacqueline F.;Mohr Judy M.;McDermott Will & Emery LLP
主权项 1. A method of treating Retinitis Pigmentosa (RP), comprising: administering an effective amount of a synthetic retinyl ester that is a 9-cis-retinyl ester or an 11-cis-retinyl ester to a human subject, wherein RP in the human subject is due to a deficiency in endogenous 11-cis retinal in the eye and wherein when the synthetic retinyl ester is administered to the human subject a functional opsin/chromophore complex is formed in the eye, wherein the 9-cis-retinyl ester has a structure as represented by Formula I or the 11-cis-retinyl ester has a structure as represented by Formula II, wherein A in each formula is CH2OR and R forms a retinyl ester, and the ester substituent of said retinyl ester comprises a carboxylate radical of a C1 to C10 monocarboxylic acid or a C2 to C22 polycarboxylic acid: wherein the ester substituent is selected from the group consisting of formate, acetate, propionate, butyrate, valerate, caproate, heptanoate, caprylate, pelargonate, caprate, oxalate, malonate, succinate, fumarate, malate, glutarate, adipate, pimelate, suberate, azelate, sebacate, citrate, oxaloacetate, and ketoglutarate with the proviso that if the synthetic retinyl ester is an 11-cis-retinyl ester it is not 11-cis-retinyl acetate.
地址 Seattle WA US