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    MRNA THERAPY FOR PHENYLKETONURIA
    2.
    发明公开
    MRNA THERAPY FOR PHENYLKETONURIA 审中-公开
    MRNA-THERAPIEFÜRPHENYLKETONURIE

    公开(公告)号：EP3060258A1

    公开(公告)日：2016-08-31

    申请号：EP14792715.6

    申请日：2014-10-22

    申请人： Shire Human Genetic Therapies, Inc.

    发明人： DEROSA, Frank ,  HEARTLEIN, Michael ,  DIAS, Anusha

    IPC分类号： A61K48/00

    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，其包括以有效剂量给药需要治疗的受试者，所述组合物包含编码苯丙氨酸羟化酶（PAH）的mRNA和施用间隔，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包封在脂质体中，所述脂质体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

    Hybrid molecules having translocation region and cell-binding region
    6.
    发明公开
    Hybrid molecules having translocation region and cell-binding region 失效
    Hybridmolekülemit Translokations- und zellbindenden Regionen。

    公开(公告)号：EP0439954A2

    公开(公告)日：1991-08-07

    申请号：EP90314221.4

    申请日：1990-12-24

    申请人： SERAGEN, INC.

    发明人： Murphy, John R.

    IPC分类号： C12N15/62 ,  C12N15/26 ,  C12N15/31 ,  C12N15/48 ,  C12N15/57 ,  A61K48/00 ,  C12N15/16

    CPC分类号： C07K14/005 ,  C07K14/245 ,  C07K14/25 ,  C07K14/28 ,  C07K14/34 ,  C07K14/415 ,  C07K14/55 ,  C07K14/575 ,  C07K2319/00 ,  C07K2319/55 ,  C07K2319/75 ,  C12N9/0071 ,  C12N9/2408 ,  C12N15/62 ,  C12N2740/16222 ,  C12N2770/24022 ,  C12Y114/16001 ,  G01N33/56966 ,  G01N33/68

    摘要： A hybrid molecule including a first part, a second part and a third part connected by covalent bonds,

    (a) the first part including a portion of the binding domain of a cell-binding ligand, which portion is able to cause the hybrid molecule of the invention to bind to an animal cell;
    (b) the second part including a portion of a translocation domain of a protein, which portion is capable of translocating the third part across the cytoplasmic membrane of the cell; and
    (c) the third part including a chemical entity to be introduced into the cell, provided that (i) the hybrid molecule is produced by expression of a recombinant DNA molecule encoding the hybrid molecule, and (ii) the second and third parts are not segments of the same naturally-occurring polypeptide toxin;
    a recombinant DNA molecule encoding the hybrid molecule; and
    a method of producing the hybrid molecule.

    摘要翻译： 包括通过共价键连接的第一部分，第二部分和第三部分的杂化分子，（a）第一部分包括细胞结合配体的结合结构域的一部分，该部分能够引起杂合分子 本发明结合动物细胞; （b）第二部分包括蛋白质的易位结构域的一部分，该部分能够将第三部分转移穿过细胞的细胞质膜; （c）第三部分包括要引入细胞的化学实体，条件是（i）通过表达编码杂合分子的重组DNA分子来产生杂交分子，和（ii）第二和第三部分是 不是相同天然存在的多肽毒素的片段; 编码该杂合分子的重组DNA分子; 和杂交分子的制造方法。

    Composition of erythrocytes encapsulating phenylalanine hydroxylase and therapeutic use thereof
    10.
    发明公开
    Composition of erythrocytes encapsulating phenylalanine hydroxylase and therapeutic use thereof 审中-公开
    该组合物是基于，封装在在苯丙氨酸羟化酶红细胞，和治疗及其用途

    公开(公告)号：EP2813234A1

    公开(公告)日：2014-12-17

    申请号：EP13305786.9

    申请日：2013-06-11

    申请人： Erytech Pharma

    发明人： Godfrin, Yann ,  Dufour, Emmanuelle ,  Cheng, Seng H. ,  Yew, Nelson S.

    IPC分类号： A61K38/00 ,  A61K38/48 ,  C12N9/02 ,  A61K9/50 ,  G01N33/556 ,  A61K9/00

    CPC分类号： A61K38/44 ,  A61K9/0019 ,  A61K9/5068 ,  A61K38/443 ,  C12N9/0071 ,  C12Y114/16001

    摘要： The present invention relates to Enzyme Replacement Therapy (ERT) based on phenylalanine hydroxylase (PAH) and compositions intended for this use. It concerns an erythrocyte encapsulating PAH, especially in suspension in a pharmaceutically acceptable carrier or vehicle, a pharmaceutical composition comprising erythrocytes encapsulating PAH in a pharmaceutically acceptable carrier or vehicle, and such a pharmaceutical composition for use in the treatment or prevention of phenylketonuria (PKU) and/or other diseases involving a too high level of phenylalanine; the treatment or prevention may be in combination with a Phe-restricted diet. The invention particularly relates to classic PKU, variant PKU and non-PKU hyperphenylalaninemia.

    摘要翻译： 本发明涉及基于苯丙氨酸羟化酶（PAH）和组合物用于此用途酶替代疗法（ERT）。 它涉及对红细胞封装PAH，爱特别是在悬浮在药学上可接受的载体或媒介物，包含红细胞在药学上可接受的载体或运载体包封PAH的药物组合物，并且这样的药物组合物用于治疗或预防苯丙酮尿症的使用（PKU） 和/或其它疾病涉及苯丙氨酸的过高的水平; 治疗或预防可以是与苯丙氨酸限制性饮食组合。 本发明特别涉及到经典PKU，变体PKU和非PKU高苯丙氨酸血症。