公开(公告)号：EP3548620A1

公开(公告)日：2019-10-09

申请号：EP17877203.4

申请日：2017-12-01

申请人： Cold Spring Harbor Laboratory ,  Ionis Pharmaceuticals, Inc.

发明人： YU, Allen, Tingjin ,  SPECTOR, David, L. ,  RIGO, Frank ,  FREIER, Susan, M. ,  BERMANN, Jan ,  BERASAIN, Carmen

IPC分类号： C12N15/113 ,  C12N15/11

公开(公告)号：EP3485015A1

公开(公告)日：2019-05-22

申请号：EP17828626.6

申请日：2017-07-17

申请人： Ionis Pharmaceuticals, Inc.

发明人： RIGO, Frank ,  PRAKASH, Thazha, P. ,  SETH, Punit, P.

IPC分类号： C12N15/113

公开(公告)号：EP3443094A1

公开(公告)日：2019-02-20

申请号：EP17783110.4

申请日：2017-04-13

申请人： Ionis Pharmaceuticals, Inc. ,  LUDWIG INSTITUTE FOR CANCER RESEARCH

发明人： BENNETT, C., Frank ,  RIGO, Frank ,  CLEVELAND, Don, W. ,  JIANG, Jie ,  ZHU, Qiang ,  LAGIER-TOURENE, Clotilde

IPC分类号： C12N15/113 ,  C12N15/11 ,  C12N15/09

公开(公告)号：EP4458361A2

公开(公告)日：2024-11-06

申请号：EP24188665.4

申请日：2013-06-25

申请人： Ionis Pharmaceuticals, Inc. ,  Baylor College Of Medicine

发明人： RIGO, Frank ,  WARD, Amanda ,  BEAUDET, Arthur L. ,  MENG, Linyan

IPC分类号： A61K31/7088

摘要： Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.

公开(公告)号：EP4281084A1

公开(公告)日：2023-11-29

申请号：EP22743253.1

申请日：2022-01-21

申请人： Ionis Pharmaceuticals, Inc.

发明人： VALAS, Ruben, E. ,  JAFAR-NEJAD, Paymaan ,  RIGO, Frank ,  FREIER, Susan, M. ,  BUI, Huynh-Hoa ,  SINGH, Priyam

IPC分类号： A61K31/7088 ,  A61K31/712 ,  A61P21/00 ,  C12N15/113

公开(公告)号：EP4172338A2

公开(公告)日：2023-05-03

申请号：EP21833921.6

申请日：2021-06-29

申请人： Ionis Pharmaceuticals, Inc.

发明人： POWERS, Berit, Elissa ,  FREIER, Susan, M. ,  BUI, Huynh-Hoa ,  RIGO, Frank

IPC分类号： C12N15/63 ,  C07K14/47 ,  C12N15/10

公开(公告)号：EP4166667A2

公开(公告)日：2023-04-19

申请号：EP22197003.1

申请日：2014-10-11

申请人： Ionis Pharmaceuticals, Inc.

发明人： FREIER, Susan, M. ,  RIGO, Frank ,  SINGH, Priyam

IPC分类号： C12N15/11 ,  A61K48/00 ,  C07H21/02 ,  C07H21/04

摘要： Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon IB of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.

公开(公告)号：EP4110924A2

公开(公告)日：2023-01-04

申请号：EP21761623.4

申请日：2021-02-26

申请人： Ionis Pharmaceuticals, Inc.

发明人： RIGO, Frank ,  JAFAR-NEJAD, Paymaan

IPC分类号： C12N15/113 ,  C07H21/04 ,  C07H19/00

公开(公告)号：EP3770258A1

公开(公告)日：2021-01-27

申请号：EP20182895.1

申请日：2013-06-25

申请人： Ionis Pharmaceuticals, Inc. ,  Baylor College Of Medicine

发明人： RIGO, Frank ,  WARD, Amanda ,  BEAUDET, Arthur L. ,  MENG, Linyan

IPC分类号： C12N15/113 ,  A61K31/7088

摘要： Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.

公开(公告)号：EP3768694A1

公开(公告)日：2021-01-27

申请号：EP19772559.1

申请日：2019-03-22

申请人： Ionis Pharmaceuticals, Inc. ,  The Regents Of The University Of Michigan

发明人： RIGO, Frank ,  TODD, Peter ,  RODRIGUEZ, Caitlin

IPC分类号： C07H21/02 ,  C12N15/113 ,  A61K31/7105