公开(公告)号：US11091759B2

公开(公告)日：2021-08-17

申请号：US15371300

申请日：2016-12-07

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Benny Sorensen ,  Pushkal Garg ,  Gabriel Robbie

IPC: C12N15/11 ,  C12N15/113 ,  A61K31/713 ,  A61K47/54

Abstract: 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.

公开(公告)号：US20220017902A1

公开(公告)日：2022-01-20

申请号：US17375785

申请日：2021-07-14

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Benny Sorensen ,  Pushkal Garg ,  Gabriel Robbie

IPC: C12N15/113 ,  A61K31/713 ,  A61K47/54

Abstract: 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.