公开(公告)号：EP4115882A1

公开(公告)日：2023-01-11

申请号：EP22178466.3

申请日：2016-01-15

申请人： The General Hospital Corporation ,  The United States of America, as represented by The Secretary, Department of Health and Human Services

发明人： SLAUGENHAUPT, Susan, A. ,  JOHNSON, Graham ,  PAQUETTE, William, D. ,  ZHANG, Wei ,  MARUGAN, Juan

IPC分类号： A61K31/4196 ,  A61P25/28 ,  C07D251/00 ,  C07D339/06

摘要： Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.

公开(公告)号：EP3244891A1

公开(公告)日：2017-11-22

申请号：EP16737944.5

申请日：2016-01-15

申请人： The General Hospital Corporation ,  United States Department of Health and Human Services

发明人： SLAUGENHAUPT, Susan, A. ,  JOHNSON, Graham ,  PAQUETTE, William, D. ,  ZHANG, Wei ,  MARUGAN, Juan

IPC分类号： A61K31/4196 ,  A61P25/28 ,  C07D251/00 ,  C07D339/06

CPC分类号： C07D473/34 ,  A61P25/28 ,  C07D405/12 ,  C07D417/12 ,  C07D471/04 ,  C07D473/16 ,  C07D487/04 ,  C07D495/04 ,  C07D513/04

摘要： Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.