[EN] MODIFIED NUCLEIC ACID MOLECULES AND USES THEREOF<br/>[FR] MOLÉCULES D'ACIDE NUCLÉIQUE MODIFIÉES ET LEURS UTILISATIONS
申请人:MODERNA THERAPEUTICS INC
公开号:WO2014093924A1
公开(公告)日:2014-06-19
The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using them.
本公开提供了经修改的核苷、核苷酸和核酸,以及它们的使用方法。
FORMULATION AND DELIVERY OF MODIFIED NUCLEOSIDE, NUCLEOTIDE, AND NUCLEIC ACID COMPOSITIONS
申请人:MODERNA THERAPEUTICS, INC.
公开号:US20160038612A1
公开(公告)日:2016-02-11
The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.
MODIFIED NUCLEOSIDE, NUCLEOTIDE, AND NUCLEIC ACID COMPOSITIONS
申请人:MODERNA THERAPEUTICS
公开号:US20130156849A1
公开(公告)日:2013-06-20
The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.
The present disclosure relates to cap analogs, which can result in high levels of capping efficiency and transcription and improved translation efficiencies. The present disclosure also relates to methods useful for preparing cap analogs and using mRNA species containing such analogs, as well as kits containing the novel cap analogs.
The present disclosure in part provides compounds (i.e., PEG lipids) which are useful in pharmaceutical compositions, cosmetic compositions, and drug delivery systems, e.g, for use in lipid nanoparticle (LNP) formulations. The present disclosure also provides LNP formulations comprising PEG lipids described herein, and methods of using the same. For example, the LNPs provided herein are useful for the delivery of an agent (e.g, therapeutic agent) to a subject. The PEG lipids and LNPs provided herein, in certain embodiments, exhibit increased PEG shedding compared to existing PEG lipids and LNP formulations.