专利检索 ap:("Hoffmann-La Roche Inc.") AND inv:"Mads Manso" 第 1 页

1.

发明申请
Antisense Oligonucleotides Targeting ATXN3 有权

公开(公告)号：US20230054720A1

公开(公告)日：2023-02-23

申请号：US17540495

申请日：2021-12-02

申请人： Hoffmann-La Roche Inc.

发明人： Heidi Rye Hudlebusch , Lykke Pedersen , Erik Funder , Lukasz Kielpinski , Christoffer Sondergaard , Alexander Herbert Stephan , Mads Manso

IPC分类号： A61K31/7088 , C12N15/113 , A61P25/28

摘要： The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia