发明名称 Agents for improved delivery of nucleic acids to eukaryotic cells
摘要 New cationic lipids are provided that are useful for delivering macromolecules, such as nucleic acids, into eukaryotic cells. The lipids can be used alone, in combination with other lipids and/or in combination with other transfection enhancing reagents to prepare transfection complexes.
申请公布号 US9259475(B2) 申请公布日期 2016.02.16
申请号 US201314054825 申请日期 2013.10.16
申请人 Molecular Transfer, Inc. 发明人 Jessee Joel;Gebeyehu Gulilat
分类号 A61K9/127;C12N15/00;C07H21/04;C07H21/02;A61K47/48;A61K31/195;C07C237/10;C07D233/64;C07C217/08;C12N15/88 主分类号 A61K9/127
代理机构 Perkins Coie LLC 代理人 Perkins Coie LLC
主权项 1. A compound having the structure: wherein each R1 is C12-C20 alkyl or monounsaturated C12-C20 alkenyl; wherein each Rx is independently C1-C3 alkyl, and each Ry is independently is selected from the group consisting of H, —CH2—(CHR3)1-6—CH2—NHR4, —CH2CH(NH2)CH2OH, and —CO—CH(R13)NR14R15, wherein each R3 independently is H, OH, or NH2, R4 is H or CH3 each R13 independently is H, C1-C6 alkyl, C1-C6 alkenyl, C2-C6 alkynyl, C3-C7 cycloalkyl, C3-C7 cycloalkyl-C1-C6 alkyl, C5-C7-cycloalkenyl, C5-C7-cycloalkenyl-C1-C6 alkyl, —(CH2)mNR6(CH2)nNHR7, —(CH2)2-6NHR7, —(CH2)3-6NHC(═NH)NH2, ((CH2)mOx)y(CH2)zOxR8, or —(CH2)0-3Het; each R14 independently is H, C1-C6 alkyl, or C1-C6 alkenyl; each R15 independently is H, an amine protecting group, —(CH2)mNR6(CH2)nNHR7, —(CH2)2-6NHR7, —(CH2)3-6NHC(═NH)NH2, —(CO)C1-C23 alkyl, —(CO)C1-C23 alkenyl, or a peptide containing 1-20 amino acid residues; each m independently is 2-5, each n independently is 2-5, each x independently is 0 or 1, y is 0-2, z is 1-6 R6 and R7 independently are H, an amine protecting group, —(CO)C1-C23 alkyl, or —(CO)C1-C23 alkenyl; R8 is H, C1-C6 alkyl, or C1-C6 alkenyl and Het is a 5-7 membered monocyclic basic heterocycle, or an 8-11 membered bicyclic basic heterocycle.
地址 Gaithersburg MD US