公开(公告)号：US11633483B2

公开(公告)日：2023-04-25

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： C07H21/02 ,  C07H21/04 ,  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：US20220380773A1

公开(公告)日：2022-12-01

申请号：US17424672

申请日：2020-01-29

申请人： Ionis Pharmaceuticals, Inc.

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

IPC分类号： 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. 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：US20220315927A1

公开(公告)日：2022-10-06

申请号：US17527953

申请日：2021-11-16

申请人： Ionis Pharmaceuticals, Inc.

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

IPC分类号： C12N15/113 ,  A61P35/00 ,  A61K9/00 ,  A61K45/00

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1.

公开(公告)号：US20210207146A1

公开(公告)日：2021-07-08

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：US20210180057A1

公开(公告)日：2021-06-17

申请号：US16759908

申请日：2018-10-31

申请人： Ionis Pharmaceuticals, Inc.

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

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

摘要： 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：US20210038631A1

公开(公告)日：2021-02-11

申请号：US16977260

申请日：2019-03-01

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： A61K31/7125 ,  C12N15/11

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：US20190233827A1

公开(公告)日：2019-08-01

申请号：US16125159

申请日：2018-09-07

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K38/36 ,  A61K31/7088 ,  C07H21/04

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：US20180311365A1

公开(公告)日：2018-11-01

申请号：US15933739

申请日：2018-03-23

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 ,  A61K31/7115 ,  A61K47/68 ,  A61P3/06 ,  A61K9/00

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：US20170166899A1

公开(公告)日：2017-06-15

申请号：US15307990

申请日：2015-05-01

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

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3525

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

公开(公告)号：US20220243203A1

公开(公告)日：2022-08-04

申请号：US17613183

申请日：2020-05-28

申请人： Ionis Pharmaceuticals, Inc.

发明人： Huynh-Hoa Bui

IPC分类号： C12N15/113

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FUS RNA in a cell or subject, and in certain instances reducing the amount of FUS protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative condition. Such symptoms and hallmarks include muscle weakness and fatigue, protein aggregates in the central nervous system, and speech difficulties and behavioral abnormalities. Non-limiting examples of neurodegenerative conditions that benefit from these compounds, methods, and pharmaceutical compositions are amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD).