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).