公开(公告)号：US20210077630A1

公开(公告)日：2021-03-18

申请号：US16953704

申请日：2020-11-20

申请人： Ionis Pharmaceuticals, Inc.

发明人： Eric E. Swayze ,  Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： A61K47/54 ,  A61K31/7125 ,  A61K31/712 ,  A61K9/00 ,  A61K47/68 ,  A61P3/06 ,  A61K31/7115 ,  C12N15/113

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting PCSK9 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PCSK9.

公开(公告)号：US20180094261A1

公开(公告)日：2018-04-05

申请号：US15593173

申请日：2017-05-11

申请人： Ionis Pharmaceuticals, Inc.

发明人： Holly Kordasiewicz ,  Eric E. Swayze ,  Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K31/711 ,  C07H21/02

摘要： Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

公开(公告)号：US09670492B2

公开(公告)日：2017-06-06

申请号：US14915039

申请日：2014-08-28

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  C12N15/113 ,  A61K31/7088 ,  A61K38/36 ,  C12N5/00

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3525 ,  C12N2320/31 ,  C12N2320/35 ,  C12Y304/21034

摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

公开(公告)号：US20240316092A1

公开(公告)日：2024-09-26

申请号：US18493056

申请日：2023-10-24

申请人： Ionis Pharmaceuticals, Inc. ,  Ludwig Institute For Cancer Research

发明人： Huynh-Hoa Bui ,  Don W. Cleveland ,  Ze'ev Melamed

IPC分类号： A61K31/7125 ,  C12N15/113

CPC分类号： A61K31/7125 ,  C12N15/113 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351

摘要： Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

公开(公告)号：US12042510B2

公开(公告)日：2024-07-23

申请号：US17554558

申请日：2021-12-17

申请人： Ionis Pharmaceuticals, Inc.

发明人： Tianyuan Zhou ,  Youngsoo Kim ,  Robert MacLeod ,  Huynh-Hoa Bui ,  Susan M. Freier

IPC分类号： C12N15/11 ,  A61K31/7125

CPC分类号： A61K31/7125 ,  C12N15/111

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.

公开(公告)号：US11833168B2

公开(公告)日：2023-12-05

申请号：US16973652

申请日：2019-06-14

申请人： Ionis Pharmaceuticals, Inc. ,  Ludwig Institute For Cancer Research

发明人： Huynh-Hoa Bui ,  Don W. Cleveland ,  Ze'ev Melamed

IPC分类号： C12N15/11 ,  A61K31/7125 ,  C12N15/113

CPC分类号： A61K31/7125 ,  C12N15/113 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351

摘要： Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

公开(公告)号：US20230118177A1

公开(公告)日：2023-04-20

申请号：US17688754

申请日：2022-03-07

申请人： Ionis Pharmaceuticals, Inc.

发明人： Thazha P. Prakash ,  Punit P. Seth ,  Eric E. Swayze ,  Sanjay Bhanot ,  Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113

摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).

公开(公告)号：US20230055405A1

公开(公告)日：2023-02-23

申请号：US17387727

申请日：2021-07-28

申请人： Ionis Pharmaceuticals, Inc.

发明人： Huynh-Hoa Bui ,  Susan M. Freier ,  Hien Thuy Zhao ,  Holly Kordasiewicz ,  Ruben E. Valas ,  Thazha P. Prakash ,  W. Brad Wan

IPC分类号： C07H21/02 ,  C12N15/113

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APP RNA in a cell or animal, and in certain instances reducing the amount of APP protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease or disorder. Such symptoms and hallmarks include cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances and seizures, progressive dementia, and abnormal amyloid deposits.

公开(公告)号：US11312964B2

公开(公告)日：2022-04-26

申请号：US16984887

申请日：2020-08-04

申请人： Ionis Pharmaceuticals, Inc.

发明人： Thazha P. Prakash ,  Punit P. Seth ,  Eric E. Swayze ,  Sanjay Bhanot ,  Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113

摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).

公开(公告)号：US20220025366A1

公开(公告)日：2022-01-27

申请号：US17294859

申请日：2019-11-21

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui ,  Hien Thuy Zhao

IPC分类号： C12N15/113 ,  A61K31/7088

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.