发明申请
- 专利标题: Lipid Nanoparticle Based Compositions and Methods for the Delivery of Biologically Active Molecules
- 专利标题(中): 基于脂质纳米颗粒的组合物和递送生物活性分子的方法
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申请号: US12027952申请日: 2008-02-07
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公开(公告)号: US20090048197A1公开(公告)日: 2009-02-19
- 发明人: Tongqian Chen , Chandra Vargeese , Kurt Vagle , Weimin Wang , Ye Zhang
- 申请人: Tongqian Chen , Chandra Vargeese , Kurt Vagle , Weimin Wang , Ye Zhang
- 申请人地址: US CA San Francisco
- 专利权人: SIRNA THERAPEUTICS, INC.
- 当前专利权人: SIRNA THERAPEUTICS, INC.
- 当前专利权人地址: US CA San Francisco
- 主分类号: A61K31/7088
- IPC分类号: A61K31/7088 ; C07H21/04 ; A61P43/00
摘要:
The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).
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