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.

公开(公告)号：US20230183696A1

公开(公告)日：2023-06-15

申请号：US17991955

申请日：2022-11-22

Applicant: Biogen MA Inc.

Inventor： Frank Rigo ,  C. Frank Bennett

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2320/33 ,  C12N2310/3341 ,  C12N2310/346 ,  C12N2310/343 ,  C12N2310/322

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

公开(公告)号：US20210389335A1

公开(公告)日：2021-12-16

申请号：US17365365

申请日：2021-07-01

Applicant: Biogen MA Inc.

Inventor： Frank Rigo ,  Katherine M. Bishop

IPC: G01N33/68 ,  G01N33/566 ,  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. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.