公开(公告)号：EP4424828A1

公开(公告)日：2024-09-04

申请号：EP24157647.9

申请日：2016-12-07

申请人： Genzyme Corporation

发明人： Akinc, Akin ,  Sorensen, Benny ,  Garg, Pushkal ,  Robbie, Gabriel

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P7/04

CPC分类号： C12N2320/3520130101 ,  A61K47/549 ,  A61K31/713 ,  C12N15/113 ,  C12N2310/1420130101 ,  C12N2310/31520130101 ,  C12N2310/34620130101 ,  C12N2310/35120130101 ,  A61P43/00 ,  A61P7/04

摘要： The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and to treat subjects having a Serpinc1-associated disease, e.g., a bleeding disorder, such as a hemophilia.