公开(公告)号：US20220135586A1

公开(公告)日：2022-05-05

申请号：US17430511

申请日：2020-02-10

申请人： PTC THERAPEUTICS, INC.

发明人： Nanjing ZHANG ,  Michael A. ARNOLD ,  Amal DAKKA ,  Gary Mitchell KARP ,  Tom Tuan LUONG ,  Jana NARASIMHAN ,  Nikolai A. NARYSHKIN ,  Jiashi WANG ,  Xiaoyan ZHANG

IPC分类号： C07D495/04 ,  A61P25/00

摘要： The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

公开(公告)号：US20230057568A1

公开(公告)日：2023-02-23

申请号：US17783941

申请日：2020-12-07

申请人： PTC THERAPEUTICS, INC.

发明人： Nanjing ZHANG ,  Michael A. ARNOLD ,  Amal DAKKA ,  Gary Mitchell KARP ,  Tom Tuan LUONG ,  Christie MORRILL ,  Jana NARASIMHAN ,  Nikolai A. NARYSHKIN ,  Anthony TURPOFF ,  Jiashi WANG ,  Xiaoyan ZHANG

IPC分类号： C07D495/04

摘要： The present description relates to compounds useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno [3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

公开(公告)号：US20220135568A1

公开(公告)日：2022-05-05

申请号：US17430566

申请日：2020-02-10

申请人： PTC THERAPEUTICS, INC.

发明人： Jiashi WANG ,  Michael A. ARNOLD ,  Amal DAKKA ,  Gary Mitchell KARP ,  Jana NARASIMHAN ,  Nikolai A. NARYSHKIN ,  Nanjing ZHANG

IPC分类号： C07D487/04

摘要： The present description relates to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect described herein relates to substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.