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    METHODS FOR THERAPEUTIC ADMINISTRATION OF MESSENGER RIBONUCLEIC ACID DRUGS
    2.
    发明公开
    METHODS FOR THERAPEUTIC ADMINISTRATION OF MESSENGER RIBONUCLEIC ACID DRUGS 审中-公开

    公开(公告)号：EP3359670A1

    公开(公告)日：2018-08-15

    申请号：EP16782372.3

    申请日：2016-10-05

    申请人： Modernatx, Inc.

    发明人： HOGE, Stephen ,  CHAKRABORTY, Tirtha ,  BESIN, Gilles ,  JAIN, Ruchi

    IPC分类号： C12N15/67

    摘要： The disclosure features methods of reducing or inhibiting an anti-drug antibody response in a subject, as well as methods of reducing or inhibiting unwanted immune cell activation in a subject to be treated with a messenger RNA (mRNA), comprising administering to the subject a mRNA, e.g., a chemically modified messenger RNA (mmRNA), encoding a polypeptide of interest, wherein the mRNA comprises at least one microRNA (miR) binding site for a miR expressed in immune cells, such as miR-126 binding site and/or miR-142 binding site, such that an anti-drug antibody response to the polypeptide or interest, or unwanted immune cell activation (e.g., B cell activation, cytokine secretion), is reduced or inhibited in the subject. The disclosure further provides therapeutic treatment regimens designed to reduce or inhibit AD A or unwanted immune cell activation (e.g., B cell activation, cytokine secretion) in a subject being treated with mRNA-based therapeutics.

    MULTIPARAMETRIC NUCLEIC ACID OPTIMIZATION
    7.
    发明公开
    MULTIPARAMETRIC NUCLEIC ACID OPTIMIZATION 审中-公开
    多参数核酸优化

    公开(公告)号：EP3218508A1

    公开(公告)日：2017-09-20

    申请号：EP15859969.6

    申请日：2015-11-04

    申请人： Modernatx, Inc.

    发明人： REYNDERS III, John van Wicheren ,  CHAKRABORTY, Tirtha ,  HOGE, Stephen ,  MCFADYEN, Iain, James

    IPC分类号： C12P21/00

    CPC分类号： C12P21/02 ,  A61K38/02 ,  C07K14/00 ,  C07K2319/00

    摘要： The present disclosure provides multiparametric codon optimization methods to improve at least a property in a candidate nucleic acid sequence. Such parameters include improving nucleic acid stability (e.g., mRNA stability), increasing translation efficacy in the target tissue, reducing the number of truncated proteins expressed, improving the folding or prevent misfolding of the expressed proteins, reducing toxicity of the expressed products, reducing cell death caused by the expressed products, and increasing or decreasing protein aggregation. After such optimization, the resulting optimized nucleic acid sequence has at least one optimized property with respect to the candidate nucleic acid sequence.

    摘要翻译： 本公开提供了多参数密码子优化方法以改善候选核酸序列中的至少一种性质。 这些参数包括提高核酸稳定性（例如mRNA稳定性），增加靶组织中的翻译效率，减少表达的截短蛋白质的数量，改善折叠或防止表达蛋白质的错误折叠，减少表达产物的毒性，减少细胞 由表达产物引起的死亡，以及增加或减少蛋白质聚集。 在这样的优化之后，所得到的优化的核酸序列相对于候选核酸序列具有至少一个优化的性质。

    TERMINAL MODIFICATIONS OF POLYNUCLEOTIDES
    8.
    发明公开
    TERMINAL MODIFICATIONS OF POLYNUCLEOTIDES 审中-公开
    多核苷酸的末端修饰

    公开(公告)号：EP3233132A1

    公开(公告)日：2017-10-25

    申请号：EP15871164.8

    申请日：2015-12-18

    申请人： Modernatx, Inc.

    发明人： CHAKRABORTY, Tirtha

    IPC分类号： A61K48/00

    CPC分类号： C12N15/85 ,  A61K48/00 ,  C12N2840/007

    摘要： The present invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides comprising at least one terminal modification. Described herein are polynucleotides comprising at least one terminal modification, methods, processes, kits and devices using the polynucleotides comprising at least one terminal modification in one or more untranslate regions. Such untranslated regions may be a 5′ or 3′ untranslated region.

    摘要翻译： 本发明涉及用于包含至少一个末端修饰的多核苷酸的制备，制造和治疗用途的组合物和方法。 本文描述了包含至少一个末端修饰的多核苷酸，方法，过程，试剂盒和装置，其使用在一个或多个非翻译区中包含至少一个末端修饰的多核苷酸。 这种非翻译区可以是5'或3'非翻译区。