公开(公告)号：US20230340489A1

公开(公告)日：2023-10-26

申请号：US18026772

申请日：2021-09-15

Applicant: Biogen MA Inc.

Inventor： Shih-Ching Lo ,  Alexander McCampbell ,  Maria Zavodszky ,  Edward Guilmette ,  Barret Pfeiffer

IPC: C12N15/113 ,  C12N15/86 ,  A61P25/28 ,  A61K48/00

CPC classification number: C12N15/1137 ,  C12N15/86 ,  A61P25/28 ,  A61K48/0058 ,  C12Y115/01001

Abstract: The present disclosure provides compositions and methods for treating amyotrophic lateral sclerosis (ALS). Among other things, the present disclosure provides inhibitory nucleic acids that inhibit the expression of genes that cause or are implicated in ALS pathogenesis. The present disclosure further provides recombinant adeno-associated virus (rAAV) vectors comprising inhibitory nucleic acids that inhibit the expression of genes that cause or are implicated in ALS pathogenesis.

公开(公告)号：US11474113B2

公开(公告)日：2022-10-18

申请号：US16963914

申请日：2019-01-25

Applicant: Biogen MA Inc.

Inventor： Wildon Farwell ,  John Staropoli ,  Guolin Zhao ,  Alexander McCampbell ,  Christopher Cody Stebbins

IPC: G01N33/68 ,  C12N15/113

Abstract: Featured are biomarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.

公开(公告)号：US20220280548A1

公开(公告)日：2022-09-08

申请号：US17635363

申请日：2020-08-14

Applicant: Biogen MA Inc.

Inventor： Alexander McCampbell

IPC: A61K31/7125 ,  A61K48/00 ,  A61P21/00 ,  C12N15/113

Abstract: Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a small molecule that promotes SMN function and/or a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and/or an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and promotes the inclusion of exon 7 in SMN2 mRNA).

公开(公告)号：US20210308281A1

公开(公告)日：2021-10-07

申请号：US17268390

申请日：2019-08-15

Applicant: Biogen MA Inc.

Inventor： Anindya Kumar Sen ,  Alexander McCampbell

IPC: A61K48/00 ,  C12N15/113 ,  A61P21/00

Abstract: Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and that promotes the inclusion of exon 7 in SMN2 mRNA).

公开(公告)号：US20210041459A1

公开(公告)日：2021-02-11

申请号：US16963914

申请日：2019-01-25

Applicant: Biogen MA Inc.

Inventor： Wildon Farwell ,  John Staropoli ,  Guolin Zhao ,  Alexander McCampbell ,  Christopher Cody Stebbins

IPC: G01N33/68 ,  C12N15/113

Abstract: Featured are biomarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.