公开(公告)号：US11781135B2

公开(公告)日：2023-10-10

申请号：US16298607

申请日：2019-03-11

申请人： Washington University ,  Biogen MA Inc.

发明人： Timothy M. Miller ,  Sarah Devos ,  C. Frank Bennett ,  Frank Rigo

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

CPC分类号： C12N15/113 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Also disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.

公开(公告)号：US10436802B2

公开(公告)日：2019-10-08

申请号：US15509642

申请日：2015-09-11

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  Katherine M. Bishop

IPC分类号： A61K48/00 ,  G01N33/68 ,  G01N33/566 ,  C12N15/113

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. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.

公开(公告)号：US20190030058A1

公开(公告)日：2019-01-31

申请号：US15892560

申请日：2018-02-09

申请人： Biogen MA 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： A61K31/7088 ,  C12N15/113 ,  A61K31/712

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.

公开(公告)号：US20200376018A1

公开(公告)日：2020-12-03

申请号：US16708739

申请日：2019-12-10

申请人： Biogen MA 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： A61K31/7088 ,  C12N15/113

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.

公开(公告)号：US20190040384A1

公开(公告)日：2019-02-07

申请号：US15907970

申请日：2018-02-28

申请人： Biogen MA Inc.

发明人： C. Frank Bennett ,  Gene Hung ,  Frank Rigo

IPC分类号： C12N15/113 ,  A61K38/30 ,  A61B5/04 ,  A61B5/0488 ,  A61K9/00 ,  A61K31/185 ,  A61K31/7088 ,  A61K35/545 ,  A61K45/06 ,  A61K47/46 ,  A61K48/00 ,  G01N33/50 ,  C12Q1/6883

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.

公开(公告)号：US20170363643A1

公开(公告)日：2017-12-21

申请号：US15509642

申请日：2015-09-11

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  Katherine M. Bishop

IPC分类号： G01N33/68 ,  C12N15/113

CPC分类号： G01N33/6896 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2320/32 ,  C12N2320/35 ,  G01N33/566 ,  G01N33/6854 ,  G01N33/6887 ,  G01N2333/47 ,  G01N2800/28 ,  G01N2800/2878 ,  G01N2800/52

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. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.

公开(公告)号：US12013403B2

公开(公告)日：2024-06-18

申请号：US17365365

申请日：2021-07-01

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  Katherine M. Bishop

IPC分类号： C07H21/04 ,  C12N15/113 ,  G01N33/566 ,  G01N33/68

CPC分类号： G01N33/6896 ,  C12N15/113 ,  G01N33/566 ,  G01N33/6854 ,  G01N33/6887 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2320/32 ,  C12N2320/35 ,  G01N2333/47 ,  G01N2800/28 ,  G01N2800/2878 ,  G01N2800/52

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. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.

公开(公告)号：US11535848B2

公开(公告)日：2022-12-27

申请号：US16837349

申请日：2020-04-01

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  C. Frank Bennett

IPC分类号： C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.

公开(公告)号：US20210032624A1

公开(公告)日：2021-02-04

申请号：US16837349

申请日：2020-04-01

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  C. Frank Bennett

IPC分类号： C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.

公开(公告)号：US09926559B2

公开(公告)日：2018-03-27

申请号：US14760171

申请日：2014-01-09

申请人： Biogen MA Inc.

发明人： C. Frank Bennett ,  Gene Hung ,  Frank Rigo

IPC分类号： C12N15/11 ,  C12N15/113 ,  A61B5/04 ,  A61B5/0488 ,  A61K9/00 ,  A61K31/185 ,  A61K31/7088 ,  A61K35/545 ,  A61K38/30 ,  A61K45/06 ,  A61K47/46 ,  A61K48/00 ,  C12Q1/68 ,  G01N33/50

CPC分类号： C12N15/113 ,  A61B5/04012 ,  A61B5/0488 ,  A61K9/0019 ,  A61K9/0085 ,  A61K31/185 ,  A61K31/7088 ,  A61K35/545 ,  A61K38/30 ,  A61K45/06 ,  A61K47/46 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/351 ,  C12Q1/6883 ,  C12Q2600/156 ,  G01N33/5091

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.