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    MRNA THERAPY FOR POMPE DISEASE
    32.
    发明申请
    MRNA THERAPY FOR POMPE DISEASE 审中-公开
    MRNA治疗疾病

    公开(公告)号：US20160324940A1

    公开(公告)日：2016-11-10

    申请号：US15073163

    申请日：2016-03-17

    申请人： Shire Human Genetic Therapies, Inc.

    发明人： Frank DeRosa ,  Michael Heartlein

    IPC分类号： A61K38/47 ,  A61K47/22 ,  A61K48/00 ,  A61K9/127 ,  A61K9/00

    CPC分类号： A61K38/47 ,  A61K9/0019 ,  A61K9/127 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7105 ,  A61K47/22 ,  A61K48/0033 ,  A61K48/0058 ,  C12N9/2465 ,  C12Y302/0102

    摘要： The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease 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： 本发明尤其提供了处理庞培病的方法，包括向需要治疗的受试者施用包含编码酸性α-葡萄糖苷酶（GAA）的​​mRNA的组合物，其有效剂量和给药间隔使得至少一种 庞培病的症状或特征在强度，严重程度或频率上降低或发作迟缓。 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。

    CELLS FOR PRODUCING RECOMBINANT IDURONATE-2-SULFATASE
    33.
    发明申请
    CELLS FOR PRODUCING RECOMBINANT IDURONATE-2-SULFATASE 有权
    用于生产重组蛋白二硫酸酯酶的细胞

    公开(公告)号：US20160053238A1

    公开(公告)日：2016-02-25

    申请号：US14832728

    申请日：2015-08-21

    申请人： Shire Human Genetic Therapies, Inc.

    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。

    METHODS FOR PURIFICATION OF MESSENGER RNA
    34.
    发明申请
    METHODS FOR PURIFICATION OF MESSENGER RNA 有权
    消化细胞RNA的方法

    公开(公告)号：US20150376220A1

    公开(公告)日：2015-12-31

    申请号：US14696140

    申请日：2015-04-24

    申请人： Shire Human Genetic Therapies, Inc.

    发明人： Frank DeRosa ,  Anusha Dias ,  Shrirang Karve ,  Michael Heartlein

    IPC分类号： C07H1/06 ,  C07H21/02

    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。

    IONIZABLE CATIONIC LIPIDS
    35.
    发明申请
    IONIZABLE CATIONIC LIPIDS 有权
    IONIZABLE阳离子脂肪

    公开(公告)号：US20150166462A1

    公开(公告)日：2015-06-18

    申请号：US14389023

    申请日：2013-03-29

    申请人： Shire Human Genetic Therapies. Inc.

    发明人： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

    IPC分类号： C07C211/21 ,  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。

    MRNA therapy for phenylketonuria
    40.
    发明授权
    MRNA therapy for phenylketonuria 有权
    苯丙酮尿症的MRNA治疗

    公开(公告)号：US09522176B2

    公开(公告)日：2016-12-20

    申请号：US14521323

    申请日：2014-10-22

    申请人： Shire Human Genetic Therapies, Inc.

    发明人： Frank DeRosa ,  Michael Heartlein ,  Anusha Dias

    IPC分类号： A61K48/00 ,  A61K9/127 ,  C12N15/74 ,  C12N15/11 ,  C07H21/04 ,  A61K38/44 ,  C12N9/02

    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。