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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：US20150376220A1
公开(公告)日：2015-12-31
申请号：US14696140
申请日：2015-04-24
发明人： Frank DeRosa , Anusha Dias , Shrirang Karve , Michael Heartlein
CPC分类号： C07H1/06 , C07H21/00 , C07H21/02 , C12N15/1017
摘要： The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.
摘要翻译： 本发明尤其提供了纯化信使RNA（mRNA）的方法，包括以下步骤：（a）从不纯制剂中沉淀mRNA; （b）使包含沉淀的mRNA的不纯制剂进行涉及膜过滤的纯化过程，使得沉淀的mRNA被膜捕获; 和（c）通过重新溶解mRNA从膜中洗脱捕获的沉淀的mRNA，从而得到纯化的mRNA溶液。 在一些实施方案中，涉及适用于本发明的膜过滤的净化方法是切向流过滤。
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公开(公告)号：US09522176B2
公开(公告)日：2016-12-20
申请号：US14521323
申请日：2014-10-22
发明人： Frank DeRosa , Michael Heartlein , Anusha Dias
CPC分类号： C12N9/0071 , A61K9/1272 , A61K38/44 , A61K48/00 , A61K48/0033 , A61K48/005 , C12Y114/16001
摘要： The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
摘要翻译： 本发明尤其提供了治疗苯丙酮尿症（PKU）的方法，其包括以有效剂量和给药间隔给予需要治疗的包含编码苯丙氨酸羟化酶（PAH）的mRNA的组合物的组合物，使得至少一种 PKU的症状或特征在强度，严重程度或频率上降低或发病延迟。 在一些实施方案中，将mRNA包封在包含一种或多种阳离子脂质，一种或多种非阳离子脂质，一种或多种基于胆固醇的脂质和一种或多种经PEG修饰的脂质的脂质体中。
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公开(公告)号：US20170073648A1
公开(公告)日：2017-03-16
申请号：US15349720
申请日：2016-11-11
发明人： Frank DeRosa , Michael Heartlein , Anusha Dias
CPC分类号： C12N9/0071 , A61K9/1272 , A61K38/44 , A61K48/00 , A61K48/0033 , A61K48/005 , C12Y114/16001
摘要： The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids
摘要翻译： 本发明尤其提供了治疗苯丙酮尿症（PKU）的方法，其包括以有效剂量和给药间隔给予需要治疗的包含编码苯丙氨酸羟化酶（PAH）的mRNA的组合物的组合物，使得至少一种 PKU的症状或特征在强度，严重程度或频率上降低或发病延迟。 在一些实施方案中，mRNA包封在包含一种或多种阳离子脂质，一种或多种非阳离子脂质，一种或多种基于胆固醇的脂质和一种或多种PEG修饰的脂质的脂质体中
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公开(公告)号：US20160040154A1
公开(公告)日：2016-02-11
申请号：US14775915
申请日：2014-03-14
发明人： Michael Heartlein , Frank DeRosa , Anusha Dias , Shrirang Karve
CPC分类号： C12N15/10 , C07H21/02 , C12N15/1017
摘要： The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.
摘要翻译： 本发明尤其提供了纯化信使RNA（mRNA）的方法，包括以下步骤：将包含体外合成的mRNA的不纯制剂进行变性，并通过切向纯化来自步骤（a）的不纯制剂的mRNA 流程过滤，其中从步骤（b）纯化的mRNA基本上不含用于体外合成的过早中止的RNA序列和/或酶试剂。
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公开(公告)号：US20150110858A1
公开(公告)日：2015-04-23
申请号：US14521323
申请日：2014-10-22
发明人： Frank DeRosa , Michael Heartlein , Anusha Dias
CPC分类号： C12N9/0071 , A61K9/1272 , A61K38/44 , A61K48/00 , A61K48/0033 , A61K48/005 , C12Y114/16001
摘要： The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids
摘要翻译： 本发明尤其提供了治疗苯丙酮尿症（PKU）的方法，其包括以有效剂量和给药间隔给予需要治疗的包含编码苯丙氨酸羟化酶（PAH）的mRNA的组合物的组合物，使得至少一种 PKU的症状或特征在强度，严重程度或频率上降低或发病延迟。 在一些实施方案中，mRNA包封在包含一种或多种阳离子脂质，一种或多种非阳离子脂质，一种或多种基于胆固醇的脂质和一种或多种PEG修饰的脂质的脂质体中
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公开(公告)号：US20160032356A1
公开(公告)日：2016-02-04
申请号：US14775846
申请日：2014-03-14
发明人： Michael Heartlein , Frank DeRosa , Anusha Dias
IPC分类号： C12Q1/68 , C07K14/505 , C12N9/02
CPC分类号： C12Q1/68 , C07K14/505 , C12N9/0069 , C12Q1/6823 , C12Y113/12007 , C12Q2521/327 , C12Q2525/186 , C12Q2537/113 , C12Q2565/137
摘要： The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.
摘要翻译： 本发明尤其提供了定量mRNA封闭效率的方法，特别是用于体外合成的mRNA的方法。 在一些实施方案中，所述方法包括量化盖帽的封端效率和甲基化状态的色谱方法。
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