发明名称 ENGINEERED NUCLEIC ACIDS AND METHODS OF USE THEREOF
摘要 Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful to treat or prevent diseases, disorders or conditions, or to improve a subject's heath or wellbeing.
申请公布号 US2016024171(A1) 申请公布日期 2016.01.28
申请号 US201514878286 申请日期 2015.10.08
申请人 Moderna Therapeutics, Inc. 发明人 Schrum Jason P.;Bancel Stephane;Afeyan Noubar B.;Ejebe Kenechi
分类号 C07K14/685 主分类号 C07K14/685
代理机构 代理人
主权项 1. A pharmaceutical formulation comprising: i) an effective amount of a messenger ribonucleic acid (mRNA) sequence encoding a melanocyte-stimulating hormone polypeptide, wherein the mRNA sequence is at least 90% identical to the sequence of SEQ ID NO: 19; and ii) a pharmaceutically acceptable carrier, wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof.
地址 Cambridge MA US