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.