发明名称 COMPOSITIONS AND METHODS FOR DELIVERING MESSENGER RNA
摘要 The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provided cationic lipids that are useful for preparing the compositions of the invention.
申请公布号 US2016256568(A1) 申请公布日期 2016.09.08
申请号 US201615153291 申请日期 2016.05.12
申请人 PROTIVA BIOTHERAPEUTICS, INC. 发明人 HEYES James;PALMER Lorne R.;REID Stephen P.;YAWORSKI Edward D.;MACLACHLAN Ian;WOOD Mark;MARTIN Alan D.
分类号 A61K48/00;A61K31/7105;A61K47/14;A61K47/28;A61K47/10;A61K9/50;A61K47/24 主分类号 A61K48/00
代理机构 代理人
主权项 1. A lipid particle comprising: (a) one or more mRNA molecule(s) that each encode a protein; (b) a cationic lipid or a salt thereof comprising from about 30 mol % to about 50 mol % of the total lipid present in the particle; (c) a mixture of a phospholipid and cholesterol or a derivative thereof comprising from about 47 mol % to about 69 mol % of the total lipid present in the particle; and (d) a PEG-lipid conjugate comprising from about 1 mol % to about 3 mol % of the total lipid present in the particle.
地址 Burnaby CA
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