公开(公告)号：WO2021202621A2

公开(公告)日：2021-10-07

申请号：PCT/US2021/025019

申请日：2021-03-30

申请人： NEUBASE THERAPEUTICS, INC.

发明人： LY, Danith H. ,  THADKE, Shivaji A. ,  BATWAL, Ramesh U. ,  DI CARO, Valentina ,  STEPHAN, Dietrich A. ,  SOOTER, Letha J. ,  BACKENROTH, Samuel I.

IPC分类号： C12Q1/6848 ,  C12Q1/6853 ,  A61K38/00 ,  A61K47/02 ,  A61K47/12 ,  A61K47/542 ,  A61K47/645 ,  A61K51/0491 ,  A61K9/0019 ,  A61K9/0053 ,  A61K9/0085 ,  A61K9/0095 ,  A61K9/06 ,  A61K9/08 ,  A61K9/10 ,  A61K9/107 ,  A61K9/127 ,  A61K9/2004 ,  A61K9/4841 ,  A61P25/28 ,  C07K14/003 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/3181 ,  C12N2320/34 ,  G01N30/7233 ,  G01N33/6896

摘要： The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.