公开(公告)号：US20230365965A1

公开(公告)日：2023-11-16

申请号：US18155655

申请日：2023-01-17

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Alfica Sehgal ,  Ivanka Toudjarska ,  Donald Foster ,  Stuart Milstein ,  Brian Bettencourt ,  Martin A. Maier ,  Klaus Charisse ,  Satyanarayana Kuchimanchi ,  Kallanthottathil G. Rajeev ,  Muthiah Manoharan

IPC: C12N15/113 ,  A61K31/713

CPC classification number: C12N15/113 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/335 ,  C12N2310/3515 ,  C12N2310/351 ,  C12N2320/30 ,  C12N2310/3521 ,  C12N2310/3533

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 methods of treating subjects having a bleeding disorder, such as a hemophilia.

公开(公告)号：US20200216844A1

公开(公告)日：2020-07-09

申请号：US16220157

申请日：2018-12-14

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Alfica Sehgal ,  Ivanka Toudjarska ,  Donald Foster ,  Stuart Milstein ,  Brian Bettencourt ,  Martin A. Maier ,  Klaus Charisse ,  Satyanarayana Kuchimanchi ,  Kallanthottathil G. Rajeev ,  Muthiah Manoharan

IPC: C12N15/113 ,  A61K31/713

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 methods of treating subjects having a bleeding disorder, such as a hemophilia.

公开(公告)号：US20210254063A1

公开(公告)日：2021-08-19

申请号：US17129761

申请日：2020-12-21

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Alfica Sehgal ,  Ivanka Toudjarska ,  Donald Foster ,  Stuart Milstein ,  Brian Bettencourt ,  Martin A. Maier ,  Klaus Charisse ,  Satyanarayana Kuchimanchi ,  Kallanthottathil G. Rajeev ,  Muthiah Manoharan

IPC: C12N15/113 ,  A61K31/713

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 methods of treating subjects having a bleeding disorder, such as a hemophilia.