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公开(公告)号：US10130649B2
公开(公告)日：2018-11-20
申请号：US14775818
申请日：2014-03-14
申请人： TRANSLATE BIO, INC.
IPC分类号： A61K9/127 , A61K31/7105 , A61K9/14 , A61K48/00 , A61K9/00
摘要： Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.
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公开(公告)号：US10087247B2
公开(公告)日：2018-10-02
申请号：US14775835
申请日：2014-03-14
申请人： TRANSLATE BIO, INC.
摘要： The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject. In some embodiments, the one or more mRNAs comprise a first mRNA encoding the heavy chain and a second mRNA encoding the light chain of the antibody.
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公开(公告)号：US10022455B2
公开(公告)日：2018-07-17
申请号：US15314818
申请日：2015-05-29
申请人： TRANSLATE BIO, INC.
发明人： Frank Derosa , Michael Heartlein
IPC分类号： C07D319/12 , A61K48/00 , A61K9/127 , A61K38/48 , A61K38/18
摘要： The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.
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