发明名称 Compositions and Methods for Inhibiting Expression of Transthyretin
摘要 The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
申请公布号 US2016264963(A1) 申请公布日期 2016.09.15
申请号 US201514965825 申请日期 2015.12.10
申请人 Alnylam Pharmaceuticals, Inc. 发明人 Sah Dinah Wen-Yee;Hinkle Gregory;Alvarez Rene;Milstein Stuart;Chen Qingmin
分类号 C12N15/113;A61K9/00;A61K31/713 主分类号 C12N15/113
代理机构 代理人
主权项 1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of transthyretin (TTR), wherein the dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region complementary to a part of a mRNA encoding transthyretin (TTR), wherein the region of complementarity is less than 30 nucleotides in length and the antisense strand comprises 15 or more contiguous nucleotides of an antisense strand selected from Tables 3A, 3B, 4, 6A, 6B, 7, and 16 or SEQ ID NO:170, SEQ ID NO:450, SEQ ID NO:730, or SEQ ID NO:1010.
地址 Cambridge MA US