首页> 外国专利> Pharmaceutical compositions for promoting expression of RNA-encoded proteins, use of modified RNA for the manufacture of pharmaceutical compositions, and kits of parts comprising the pharmaceutical compositions

Pharmaceutical compositions for promoting expression of RNA-encoded proteins, use of modified RNA for the manufacture of pharmaceutical compositions, and kits of parts comprising the pharmaceutical compositions

机译:用于促进RNA编码蛋白表达的药物组合物,修饰的RNA在制备药物组合物中的用途以及包括该药物组合物的试剂盒

摘要

The invention relates to an RNA comprising at least one open reading frame (ORF) and comprising at least one modification, which increases the expression of the encoded peptide or protein. Furthermore, the invention relates to the medical use of such a modified RNA administered to a subject by jet injection. The invention relates further to a pharmaceutical composition and to a kit of parts comprising said modified RNA for administration by jet injection, preferably for use in the field of gene therapy and/or genetic vaccination. Additionally, the invention relates to a method for enhancing the (localized) expression of RNA-encoded peptides or proteins in the dermis or muscle (of a mammal) comprising administering the modified RNA by jet injection. And finally, the invention relates to a method of treatment comprising administering the modified RNA by jet injection to a subject in need thereof.
机译:本发明涉及一种RNA,其包含至少一个开放阅读框(ORF)并包含至少一种修饰,其增加了所编码的肽或蛋白质的表达。此外,本发明涉及通过喷射注射施用于受试者的这种修饰的RNA的医学用途。本发明进一步涉及药物组合物和试剂盒,其包含所述修饰的RNA,用于通过喷射注射施用,优选用于基因治疗和/或基因疫苗接种领域。另外,本发明涉及用于增强(哺乳动物的)真皮或肌肉中RNA编码的肽或蛋白质的(局部)表达的方法,其包括通过喷射注射施用修饰的RNA。最后,本发明涉及一种治疗方法,该方法包括通过喷射注射将修饰的RNA施用于有此需要的受试者。

著录项

  • 公开/公告号JP6648019B2

    专利类型

  • 公开/公告日2020-02-14

    原文格式PDF

  • 申请/专利权人 キュアバック アーゲー;

    申请/专利号JP20160535369

  • 发明设计人 バウムホーフ;パトリック;

    申请日2014-08-21

  • 分类号C12N15/67;C12N15/68;C12N15/87;C12N15/13;A61K9/08;A61K48;

  • 国家 JP

  • 入库时间 2022-08-21 11:33:28

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