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公开(公告)号:US20150299181A1
公开(公告)日:2015-10-22
申请号:US14688397
申请日:2015-04-16
申请人: Gregory Raymond BEBERNITZ , Emma CODY , Tajesh PATEL , Ming QIAN , Lewis WHITEHEAD , Thomas ZABAWA , Frederic ZECRI
发明人: Gregory Raymond BEBERNITZ , Emma CODY , Tajesh PATEL , Ming QIAN , Lewis WHITEHEAD , Thomas ZABAWA , Frederic ZECRI
IPC分类号: C07D417/12 , A61K31/41 , C07D405/14 , A61K31/428 , A61K31/343 , C07D407/12 , A61K31/357 , A61K31/443 , C07D405/04 , C07D307/85
CPC分类号: C07D405/04 , A61K31/343 , A61K31/357 , A61K31/36 , A61K31/41 , A61K31/428 , A61K31/443 , A61K31/4439 , A61K31/5377 , C07D307/85 , C07D405/12 , C07D405/14 , C07D407/12 , C07D417/12 , C07D417/14
摘要: The present invention provides a compound of formula I, in which R1, R2, X and R3 are defined in the Summary of the Invention, or a pharmaceutically acceptable salt thereof; a method for manufacturing the compounds of the invention, and its therapeutic uses. The present invention further provides a combination of pharmacologically active agents and a pharmaceutical composition.
摘要翻译: 本发明提供式I化合物或其药学上可接受的盐,其中R 1,R 2,X和R 3在发明概述中定义。 制备本发明化合物的方法及其治疗用途。 本发明还提供药理活性剂和药物组合物的组合。
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公开(公告)号:US20140303232A1
公开(公告)日:2014-10-09
申请号:US14200359
申请日:2014-03-07
申请人: Jeremy Lee BARYZA , Rohan Eric John BECKWITH , Keith BOWMAN , Crystal BYERS , Tanzina FAZAL , Gabriel Grant GAMBER , Cameron Chuck-munn LEE , Ritesh Bhanudasji TICHKULE , Chandra VARGEESE , Shuangxi WANG , Laura Ellen West , Thomas ZABAWA , Junping ZHAO
发明人: Jeremy Lee BARYZA , Rohan Eric John BECKWITH , Keith BOWMAN , Crystal BYERS , Tanzina FAZAL , Gabriel Grant GAMBER , Cameron Chuck-munn LEE , Ritesh Bhanudasji TICHKULE , Chandra VARGEESE , Shuangxi WANG , Laura Ellen West , Thomas ZABAWA , Junping ZHAO
IPC分类号: A61K47/18 , A61K47/12 , A61K47/14 , C12N15/113 , A61K47/22
CPC分类号: A61K47/18 , A61K9/1271 , A61K9/1272 , A61K47/183 , A61K47/22 , C07C217/58 , C07C217/60 , C07C217/62 , C07C219/22 , C07C219/28 , C07C229/12 , C07C235/46 , C07C237/08 , C07D205/04 , C07D213/69 , C07D213/79 , C07D295/096 , C07D317/24 , C07D317/28 , C12N15/1137 , C12N2310/14 , C12N2320/32
摘要: This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.
摘要翻译: 本发明提供式(I)化合物或其药学上可接受的盐,其中R 1 -R 4,L和X如本文所定义。 式(I)化合物及其药学上可接受的盐是可用于将生物活性剂递送至细胞和组织的阳离子脂质。
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公开(公告)号:US20160367638A1
公开(公告)日:2016-12-22
申请号:US15104843
申请日:2014-12-17
申请人: Crystal BYERS , Shari Lynn CAPLAN , Gabriel Grant GAMBER , Seung HAHM , Kurt Alex HELDWEIN , Igor SPLAWSKI , Thomas ZABAWA , Frédéric ZECRI
发明人: Crystal BYERS , Shari Lynn CAPLAN , Gabriel GAMBERS , Seung HAHM , Kurt Alex HELDWEIN , Igor SPLAWSKI , Thomas ZABAWA , Frédéric ZECRI
CPC分类号: A61K38/2264 , A61K9/0019 , A61K9/1617 , A61K9/1641 , A61K9/5123 , A61K31/7105 , A61K47/543 , A61K48/00 , A61K48/0025 , A61K48/005 , A61K48/0075 , A61K48/0091
摘要: A formulation comprising a modified synthetic messenger RNA and a delivery agent. The modified synthetic messenger RNA encodes a leptin protein, is non-replicating and is translation-ready. The formulation can be delivered to a subject with congenital leptin deficiency, lipodystrophy or other condition where circulating leptin level is low.
摘要翻译: 包含修饰的合成信使RNA和递送试剂的制剂。 修饰的合成信使RNA编码瘦素蛋白,是非复制型并且是翻译准备的。 该制剂可以递送到具有先天性瘦素缺乏症,脂肪营养不良或循环瘦素水平低的其他病症的受试者。
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