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：US20190040384A1

公开(公告)日：2019-02-07

申请号：US15907970

申请日：2018-02-28

Applicant: Biogen MA Inc.

Inventor： C. Frank Bennett ,  Gene Hung ,  Frank Rigo

IPC: 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

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：US09926559B2

公开(公告)日：2018-03-27

申请号：US14760171

申请日：2014-01-09

Applicant: Biogen MA Inc.

Inventor： C. Frank Bennett ,  Gene Hung ,  Frank Rigo

IPC: 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 classification number: 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

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：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：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.