发明名称 Ionizable cationic lipids
摘要 Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
申请公布号 US9546128(B2) 申请公布日期 2017.01.17
申请号 US201314389023 申请日期 2013.03.29
申请人 Shire Human Genetic Therapies, Inc. 发明人 DeRosa Frank;Guild Braydon Charles;Heartlein Michael
分类号 C07C229/00;C07C211/21;A61K9/127;C12N15/85 主分类号 C07C229/00
代理机构 Proskauer Rose LLP 代理人 Proskauer Rose LLP ;Chen Fangli
主权项 1. A compound having the structure: wherein R1 and R2 are each independently selected from the group consisting of hydrogen, an optionally substituted, variably saturated or unsaturated C1-C20 alkyl and an optionally substituted, variably saturated or unsaturated C6-C20 acyl; L1 and L2 are each independently optionally substituted polyunsaturated C6-C20 alkenyl; m and o are each independently selected from the group consisting of zero and any positive integer; and n is any positive integer.
地址 Lexington MA US