公开(公告)号：US10577607B2

公开(公告)日：2020-03-03

申请号：US16085140

申请日：2017-03-16

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Brett P. Monia ,  Shuling Guo ,  Susan F. Murray

IPC: C12N15/113 ,  A61K31/7125 ,  A61P3/04 ,  A61K31/712 ,  A61K31/7115 ,  A61K9/00 ,  A61K31/7088 ,  A61K31/713 ,  A61K45/06

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of DYRKIB in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a metabolic disease or disorder in an individual in need.

公开(公告)号：US20180273946A1

公开(公告)日：2018-09-27

申请号：US15729860

申请日：2017-10-11

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Brett P. Monia ,  Susan M. Freier ,  Andrew M. Siwkowski ,  Shuling Guo

IPC: C12N15/113 ,  A61K31/712

CPC classification number: C12N15/113 ,  A61K31/712 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/52 ,  C12N2320/30 ,  C12N2310/3521

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.

公开(公告)号：US20180223289A1

公开(公告)日：2018-08-09

申请号：US15850093

申请日：2017-12-21

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Shuling Guo

IPC: C12N15/113 ,  A61K45/06 ,  A61K31/713

CPC classification number: C12N15/1137 ,  A61K31/713 ,  A61K45/06 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Y304/21 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Disclosed herein are antisense compounds and methods for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as hemochromatosis or β-thalassemia can be ameliorated or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US09725722B2

公开(公告)日：2017-08-08

申请号：US14356863

申请日：2012-11-07

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Shuling Guo

IPC: A61K31/70 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  C12N5/00

CPC classification number: C12N15/1137 ,  A61K31/713 ,  A61K45/06 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Y304/21 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Disclosed herein are antisense compounds and methods for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as hemochromatosis or β-thalassemia can be ameliorated or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US20240327838A1

公开(公告)日：2024-10-03

申请号：US18492683

申请日：2023-10-23

Applicant: IONIS PHARMACEUTICALS, INC.

Inventor： Jeffrey R. Crosby ,  Shuling Guo ,  Huynh-Hoa Bui ,  Andrew T. Watt ,  Susan M. Freier

IPC: C12N15/113 ,  A61K9/00 ,  A61K31/404 ,  A61K31/47 ,  A61K31/7105 ,  A61K31/713 ,  A61M11/00 ,  A61P11/00

CPC classification number: C12N15/113 ,  A61K9/0019 ,  A61K9/0075 ,  A61K9/0078 ,  A61K31/404 ,  A61K31/47 ,  A61K31/7105 ,  A61K31/713 ,  A61P11/00 ,  A61M11/005 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting ENaC expression, which may be useful for treating, preventing, or ameliorating a disease associated with ENaC.

公开(公告)号：US20240026364A1

公开(公告)日：2024-01-25

申请号：US18358591

申请日：2023-07-25

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Shuling Guo ,  Mariam Aghajan ,  Eric E. Swayze

IPC: C12N15/113 ,  A61K47/54 ,  C07H21/00 ,  A61P7/06 ,  A61P7/00

CPC classification number: C12N15/1137 ,  A61K47/549 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1138 ,  A61P7/06 ,  A61P7/00 ,  C12Y304/21109 ,  C12N2310/11 ,  C12N2310/351 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/334 ,  C12N2310/341 ,  C12N2310/3525 ,  C12N2310/3527

Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or β-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US20220259600A1

公开(公告)日：2022-08-18

申请号：US17484386

申请日：2021-09-24

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Shuling Guo ,  Mariam Aghajan ,  Eric E. Swayze

IPC: C12N15/113 ,  A61K47/54 ,  C07H21/00 ,  A61P7/06 ,  A61P7/00

Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or β-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US20210180057A1

公开(公告)日：2021-06-17

申请号：US16759908

申请日：2018-10-31

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Jeffrey R. Crosby ,  Shuling Guo ,  Huynh-Hoa Bui ,  Andrew T. Watt ,  Susan M. Freier

IPC: C12N15/113 ,  A61K9/00 ,  A61P11/00 ,  A61K31/404 ,  A61K31/47 ,  A61K31/7105 ,  A61K31/713

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting ENaC expression, which may be useful for treating, preventing, or ameliorating a disease associated with ENaC.

公开(公告)号：US10184125B2

公开(公告)日：2019-01-22

申请号：US15850093

申请日：2017-12-21

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Shuling Guo

IPC: C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  C12N15/113 ,  A61K31/713 ,  A61K45/06 ,  C12N5/00

Abstract: Disclosed herein are antisense compounds and methods for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as hemochromatosis or β-thalassemia can be ameliorated or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US20170335322A1

公开(公告)日：2017-11-23

申请号：US15526447

申请日：2015-11-12

Applicant: Ionis Pharmaceuticals, Inc. ,  Board of Regents of the University of Texas System

Inventor： Shuling Guo ,  Punit P. Seth ,  Jacqueline T. Hecht ,  Karen LaShea Posey

IPC: C12N15/113 ,  A61K31/232 ,  A61K31/7076 ,  A61K31/405 ,  A61K45/06 ,  A61K31/192 ,  A61K31/713 ,  A61K31/235

CPC classification number: C12N15/113 ,  A61K31/12 ,  A61K31/192 ,  A61K31/232 ,  A61K31/235 ,  A61K31/405 ,  A61K31/7076 ,  A61K31/7125 ,  A61K31/713 ,  A61K45/06 ,  C12N15/00 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/341 ,  C12N2320/31 ,  A61K2300/00

Abstract: In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, the target nucleic acid is a nucleic acid that encodes cartilage oligomeric matrix protein. In certain embodiments, the target nucleic acid is a nucleic acid expressed in the growth plate, tendon, or cartilage.