公开(公告)号：US20220143066A1

公开(公告)日：2022-05-12

申请号：US17348125

申请日：2021-06-15

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

发明人： David L. Spector ,  Sarah Daniela Diermeier ,  Frank Rigo ,  C. Frank Bennett ,  Susan M. Freier ,  Gayatri Arun ,  Kung-Chi Chang

IPC分类号： A61K31/713 ,  A61P35/00 ,  A61K31/175 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/04 ,  C12N15/113

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

公开(公告)号：US11236330B2

公开(公告)日：2022-02-01

申请号：US16149941

申请日：2018-10-02

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

发明人： Frank Rigo ,  C. Frank Bennett ,  Adrian R. Krainer ,  Zhenxun Wang

IPC分类号： C07H21/04 ,  C12N15/113

摘要： The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.

公开(公告)号：US11066668B2

公开(公告)日：2021-07-20

申请号：US16422128

申请日：2019-05-24

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

发明人： C. Frank Bennett ,  Frank Rigo ,  Adrian R. Krainer ,  Rahul Sinha

IPC分类号： C12N15/113 ,  C12N15/11

摘要： The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

公开(公告)号：US11058709B1

公开(公告)日：2021-07-13

申请号：US15781249

申请日：2016-12-05

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

发明人： David L. Spector ,  Sarah Daniela Diermeier ,  Frank Rigo ,  C. Frank Bennett ,  Susan M. Freier ,  Gayatri Arun ,  Kung-Chi Chang

IPC分类号： A61K31/713 ,  A61P35/00 ,  A61K31/175 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/04 ,  C12N15/113

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

公开(公告)号：US20200032260A1

公开(公告)日：2020-01-30

申请号：US16422128

申请日：2019-05-24

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

发明人： C. Frank Bennett ,  Frank Rigo ,  Adrian R. Krainer ,  Rahul Sinha

IPC分类号： C12N15/113 ,  C12N15/11

摘要： The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

公开(公告)号：US20180371457A1

公开(公告)日：2018-12-27

申请号：US15993090

申请日：2018-05-30

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

发明人： C. Frank Bennett ,  Frank Rigo ,  Adrian R. Krainer ,  Rahul Sinha

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/33

摘要： The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

公开(公告)号：US11033570B2

公开(公告)日：2021-06-15

申请号：US16465083

申请日：2017-12-01

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 ,  C12N15/113 ,  A61K9/00 ,  A61K31/7125 ,  A61K45/06

摘要： Provided herein are methods, compounds, and compositions for reducing expression of Inc05 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：US20170015995A1

公开(公告)日：2017-01-19

申请号：US14617388

申请日：2015-02-09

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

发明人： C. Frank Bennett ,  Gene Hung ,  Frank Rigo ,  Adrian R. Krainer ,  Yimin Hua ,  Marco A. Passini ,  Lamya Shihabuddin ,  Seng H. Cheng ,  Katherine W. Klinger

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K31/7088

CPC分类号： A61K31/7088 ,  A61K31/712 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/33

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

摘要翻译： 本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。

公开(公告)号：US20190249175A1

公开(公告)日：2019-08-15

申请号：US16149941

申请日：2018-10-02

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

发明人： Frank Rigo ,  C. Frank Bennett ,  Adrian R. Krainer ,  Zhenxun Wang

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12Y207/0104

摘要： The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.

公开(公告)号：US20190211330A1

公开(公告)日：2019-07-11

申请号：US16178129

申请日：2018-11-01

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

发明人： Yimin Hua ,  Adrian R. Krainer ,  Frank Rigo ,  C. Frank Bennett

IPC分类号： C12N15/113 ,  A61K38/27 ,  A61B17/00 ,  A61K38/30

CPC分类号： C12N15/113 ,  A61B17/0057 ,  A61B2017/00455 ,  A61B2017/00575 ,  A61B2017/00601 ,  A61B2017/00623 ,  A61B2017/00676 ,  A61B2017/00893 ,  A61B2017/22069 ,  A61B2017/22072 ,  A61K38/27 ,  A61K38/30 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3525

摘要： Disclosed herein are compounds, compositions and methods for treatment of diseases and disorders, including spinal muscular atrophy.