公开(公告)号：US20220290137A1

公开(公告)日：2022-09-15

申请号：US17508516

申请日：2021-10-22

申请人： Ionis Pharmaceuticals, Inc.

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

IPC分类号： C12N15/113 ,  C07H21/04 ,  A61K9/00 ,  A61P11/00 ,  A61K47/02

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SPDEF RNA in a cell or subject, and in certain instances reducing the amount of SPDEF protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or condition characterized by excessive mucus production or fibrosis, including cystic fibrosis, asthma, chronic obstructive pulmonary disease (COPD), pulmonary fibrosis, idiopathic pulmonary fibrosis (IPF), chronic bronchitis, rhinitis and ulcerative colitis.

公开(公告)号：US20220195430A1

公开(公告)日：2022-06-23

申请号：US17393738

申请日：2021-08-04

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier

IPC分类号： C12N15/113

摘要： Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.

公开(公告)号：US20220112503A1

公开(公告)日：2022-04-14

申请号：US17416392

申请日：2019-12-20

申请人： Ionis Pharmaceuticals, Inc.

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

IPC分类号： C12N15/113 ,  A61P25/28

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 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 demyelination, progressive axonal damage and/or loss, weakness and wasting of foot and lower leg muscles, foot deformities, and weakness and atrophy in the hands. Such neurodegenerative diseases include Charcot-Marie-Tooth disease.

公开(公告)号：US20210290653A1

公开(公告)日：2021-09-23

申请号：US17337762

申请日：2021-06-03

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

发明人： Allen Tingjin Yu ,  David L. Spector ,  Frank Rigo ,  Susan M. Freier ,  Jan Bergmann ,  Carmen Berasain

IPC分类号： A61K31/713 ,  A61K9/00 ,  A61K31/7125 ,  C12N15/113

摘要： Provided herein are methods, compounds, and compositions for reducing expression of lnc05 in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a cancer in an individual.

公开(公告)号：US11072794B2

公开(公告)日：2021-07-27

申请号：US16848968

申请日：2020-04-15

申请人： IONIS PHARMACEUTICALS, Inc.

发明人： Susan M. Freier

IPC分类号： C07H21/04 ,  C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US20210147837A1

公开(公告)日：2021-05-20

申请号：US16946649

申请日：2020-06-30

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Eric E. Swayze

IPC分类号： C12N15/113 ,  C12N15/11

摘要： The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

公开(公告)号：US20210139900A1

公开(公告)日：2021-05-13

申请号：US17068185

申请日：2020-10-12

申请人： Ionis Pharmaceuticals, Inc.

发明人： Gene Hung ,  C. Frank Bennett ,  Susan M. Freier ,  Holly Kordasiewicz ,  Lisa Stanek ,  Don W. Cleveland ,  Seng H. Cheng ,  Lamya Shihabuddin

IPC分类号： C12N15/113

摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

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：US10927377B2

公开(公告)日：2021-02-23

申请号：US16418060

申请日：2019-05-21

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier

IPC分类号： C07H21/04 ,  C12N15/113 ,  A61P13/02

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.

公开(公告)号：US20200377946A1

公开(公告)日：2020-12-03

申请号：US16742320

申请日：2020-01-14

申请人： Ionis Pharmaceuticals, Inc. ,  The University of British Columbia

发明人： C. Frank Bennett ,  Michael Hayden ,  Susan M. Freier ,  Sarah Greenlee ,  Jeffrey Carroll ,  Simon Warby ,  Eric E. Swayze

IPC分类号： C12Q1/6883 ,  C12Q1/6897 ,  C12Q1/6811 ,  C12N15/113

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).