公开(公告)号：US20180291376A1

公开(公告)日：2018-10-11

申请号：US15818073

申请日：2017-11-20

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113 ,  C12N15/11

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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：US09717750B2

公开(公告)日：2017-08-01

申请号：US14617388

申请日：2015-02-09

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

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

IPC: C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  A61K31/7088 ,  C12N15/113 ,  C12Q1/68 ,  A61K31/712

CPC classification number: A61K31/7088 ,  A61K31/712 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/33

Abstract: 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：US20190030058A1

公开(公告)日：2019-01-31

申请号：US15892560

申请日：2018-02-09

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

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

IPC: A61K31/7088 ,  C12N15/113 ,  A61K31/712

Abstract: 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：US20240238326A1

公开(公告)日：2024-07-18

申请号：US18494445

申请日：2023-10-25

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

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

IPC: A61K31/7088 ,  C12N15/113 ,  A61K31/712

CPC classification number: A61K31/7088 ,  C12N15/113 ,  A61K31/712 ,  C12N2310/11 ,  C12N2320/33

Abstract: 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

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

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

IPC: A61K31/7088 ,  C12N15/113

Abstract: 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：US20170088835A1

公开(公告)日：2017-03-30

申请号：US15267408

申请日：2016-09-16

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/33 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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：US20200181613A1

公开(公告)日：2020-06-11

申请号：US16507985

申请日：2019-07-10

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113 ,  C12N15/11

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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：US20150353929A1

公开(公告)日：2015-12-10

申请号：US14584112

申请日：2014-12-29

Applicant: Isis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/33 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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.

Abstract translation: 本文公开了用于调节细胞，组织或动物中SMN2mRNA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：US08946183B2

公开(公告)日：2015-02-03

申请号：US13720474

申请日：2012-12-19

Applicant: Isis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Brenda F. Baker ,  Adrian R. Krainer ,  Yimin Hua

IPC: C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  C12N15/113 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/33 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. 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.

Abstract translation: 本文公开了用于调节细胞，组织或动物中SMN2mRNA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：US20190211330A1

公开(公告)日：2019-07-11

申请号：US16178129

申请日：2018-11-01

Applicant: Ionis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

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

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

CPC classification number: 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

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