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公开(公告)号：US09546128B2
公开(公告)日：2017-01-17
申请号：US14389023
申请日：2013-03-29
IPC分类号： C07C229/00 , C07C211/21 , A61K9/127 , C12N15/85
CPC分类号： C07C211/21 , A61K9/1271 , A61K9/1272 , A61K9/5015 , A61K38/1816 , A61K38/47 , C12N15/85 , C12Y302/01023
摘要： Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
摘要翻译： 本文公开了新化合物，包含这些化合物的药物组合物及其使用的相关方法。 本文所述的化合物是有用的，例如作为促进将包封的多核苷酸递送至靶细胞并随后转染所述靶细胞的脂质体递送载体，并且在某些实施方案中，特征在于具有一种或多种提供此类化合物相对于 其他类似分类的脂质。
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公开(公告)号：US20160122727A1
公开(公告)日：2016-05-05
申请号：US14898071
申请日：2014-06-12
发明人： Michael Heartlein , Frank DeRosa , Lianne Smith
CPC分类号： C12N7/00 , A61K35/76 , A61K38/43 , A61K48/00 , C12N15/86 , C12N2740/10033 , C12N2740/10041 , C12N2740/10052 , C12N2740/15052 , C12N2750/14133 , C12N2750/14141 , C12N2750/14152 , C12N2800/40 , C12N2999/007
摘要： The present invention provides methods for producing recombinant viral particles based on the use of exogenous mRNAs to supply various helper factors for assembly of viral particles, purified recombinant viral particles produced using such methods, and methods of using such viral particles.
摘要翻译： 本发明提供了基于使用外源mRNA供应用于装配病毒颗粒的各种辅助因子，使用这种方法产生的纯化重组病毒颗粒以及使用这种病毒颗粒的方法来生产重组病毒颗粒的方法。
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公开(公告)号：US20160031981A1
公开(公告)日：2016-02-04
申请号：US14775835
申请日：2014-03-14
CPC分类号： C07K16/24 , A61K9/0019 , A61K9/127 , A61K9/1271 , A61K9/1272 , A61K47/6911 , C07K16/22 , C07K2317/14 , C12N15/88
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.
摘要翻译： 本发明尤其提供了通过向有需要的受试者施用编码抗体的重链和轻链的一种或多种mRNA的方式和组合物，其中所述抗体以系统方式表达 主题。 在一些实施方案中，一个或多个mRNA包含编码重链的第一mRNA和编码抗体轻链的第二个mRNA。
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公开(公告)号：US20160002705A1
公开(公告)日：2016-01-07
申请号：US14775844
申请日：2014-03-14
发明人： Michael Heartlein , Frank DeRosa , Anusha Dias
IPC分类号： C12Q1/68
CPC分类号： C12Q1/6804 , C12Q2522/101 , C12Q2525/186 , C12Q2537/125 , C12Q2563/125
摘要： The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly mRNA synthesized in vitro. In some embodiments, methods according to the present invention comprise providing an mRNA sample containing capped and uncapped mRNA, providing a cap specific binding substance under conditions that permit the formation of a complex between the cap specific binding substance and the capped mRNA, and quantitatively determining the amount of the complex as compared to a control, thereby quantifying mRNA capping efficiency.
摘要翻译： 本发明尤其提供了定量mRNA封闭效率的方法，特别是在体外合成的mRNA的方法。 在一些实施方案中，根据本发明的方法包括提供含有封端和未加帽的mRNA的mRNA样品，在允许在帽特异性结合物质与加盖的mRNA之间形成复合物的条件下提供上限特异性结合物质，并定量测定 与对照相比复合物的量，从而量化mRNA封闭效率。
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公开(公告)号：US20150086613A1
公开(公告)日：2015-03-26
申请号：US14389165
申请日：2013-03-29
CPC分类号： A61K9/1277 , A61K9/1271 , A61K9/1272 , A61K38/44 , A61K47/28 , A61K48/00 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2310/141 , C12N2310/16 , C12N2310/3515 , C12N2320/32 , C12Y113/12007
摘要： Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.
摘要翻译： 本文公开了使用这些化合物制备的新型脂质和脂质体组合物以及相关方法中和或改性这些脂质体组合物。 本文所述的脂质可用作例如促进将包封的多核苷酸递送至靶细胞的脂质体载体，以及随后转染此类靶细胞。 在某些实施方案中，包含脂质体递送载体的一种或多种化合物可以被中和或进一步修饰，使得脂质体递送载体的性质被改变。
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公开(公告)号：US20150004217A1
公开(公告)日：2015-01-01
申请号：US14325594
申请日：2014-07-08
CPC分类号： A61K38/47 , A61K38/1816 , A61K38/27 , A61K38/45 , A61K48/005 , C07K14/505 , C07K14/61 , C12N9/1018 , C12N9/2465 , C12N15/67 , C12Y201/03003 , C12Y302/01022
摘要： Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.
摘要翻译： 用于调节包含至少一个RNA分子的靶细胞中蛋白质表达的组合物，其包含至少一个赋予RNA稳定性的修饰以及相关方法。
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公开(公告)号：US20140004593A1
公开(公告)日：2014-01-02
申请号：US13829780
申请日：2013-03-14
发明人： Ferenc Boldog , Michael Heartlein
IPC分类号： C12N9/16
CPC分类号： C12N9/16 , C12Y301/06013
摘要： The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co-express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-express recombinant I2S and FGE proteins. Cells according to the invention are adaptable to various cell culture conditions. In some embodiments, cells of the present invention adaptable to a large-scale suspension serum-free culture.
摘要翻译： 本发明尤其提供了使用细胞共表达I2S和FGE蛋白的生产具有改善的效力和活性的重组I2S蛋白的方法和组合物。 在一些实施方案中，根据本发明的细胞被工程化以同时过表达重组I2S和FGE蛋白。 根据本发明的细胞适用于各种细胞培养条件。 在一些实施方案中，本发明的细胞适应于大规模悬浮液无血清培养。
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公开(公告)号：US09932377B2
公开(公告)日：2018-04-03
申请号：US14993500
申请日：2016-01-12
CPC分类号： C07K14/47 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K38/00 , A61K38/1709 , A61K47/645 , A61K49/0008 , C07K7/08 , C07K2319/07 , C07K2319/10
摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
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公开(公告)号：US09713626B2
公开(公告)日：2017-07-25
申请号：US14876071
申请日：2015-10-06
发明人： Michael Heartlein , Braydon Charles Guild , Frank DeRosa , Carsten Rudolph , Christian Plank , Lianne Smith
IPC分类号： A61K9/127 , C07H21/04 , A61K31/713 , C07K14/705 , A61K31/7105 , A61K47/48 , A61K31/7115 , A61K9/00 , A61K9/51 , C12N9/14 , C12N15/63 , C07H21/02 , A61K48/00 , C07K14/47 , C12N15/88
CPC分类号： A61K31/713 , A61K9/0073 , A61K9/0078 , A61K9/127 , A61K9/1271 , A61K9/5123 , A61K31/7105 , A61K31/7115 , A61K47/6935 , A61K48/00 , A61K48/005 , C07H21/02 , C07K14/4712 , C07K14/705 , C12N9/14 , C12N15/63 , C12N15/88 , C12Y306/03049
摘要： Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1 are provided. The present invention is particularly useful for treating cystic fibrosis.
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公开(公告)号：US09629804B2
公开(公告)日：2017-04-25
申请号：US14521161
申请日：2014-10-22
发明人： Michael Heartlein , Daniel Anderson , Yizhou Dong , Frank DeRosa
CPC分类号： A61K9/1272 , A61K38/1709 , A61K38/177 , A61K38/4846 , A61K38/53 , A61K48/00 , A61K48/0033 , A61K48/005
摘要： The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c: or a pharmaceutically acceptable salt thereof.
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