公开(公告)号：US20250066389A1

公开(公告)日：2025-02-27

申请号：US18710312

申请日：2022-11-18

Applicant: Rgenta Therapeutics, Inc.

Inventor： Travis T. Wager ,  Zhiping Weng ,  Hualin Simon Xi

IPC: C07D519/00 ,  A61K31/416 ,  A61K31/437 ,  A61K31/4375 ,  A61K31/439 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/497 ,  A61K31/498 ,  A61K31/4985 ,  A61K31/4995 ,  A61K31/501 ,  A61K31/506 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/538 ,  A61K31/5386 ,  A61K31/55 ,  A61K31/551 ,  A61K31/553 ,  C07D231/56 ,  C07D401/12 ,  C07D403/12 ,  C07D403/14 ,  C07D405/14 ,  C07D409/14 ,  C07D471/04 ,  C07D487/04 ,  C07D487/10 ,  C07D491/107 ,  C07D495/04 ,  C07D498/10

Abstract: The present disclosure relates to compounds of Formula (I), and subformulas thereof, and to their pharmaceutically acceptable salts, pharmaceutical compositions, methods of use, and methods for their preparation. The compounds of the present disclosure may act as small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded genes, and methods of use of the compounds for modulating splicing and treating related diseases and conditions. The compounds disclosed herein may possess activity toward various genetic pathways and are accordingly useful in methods of treatment of diseases or disorders of the human or animal body.

公开(公告)号：US20250042879A1

公开(公告)日：2025-02-06

申请号：US18711986

申请日：2022-11-22

Applicant: Rgenta Therapeutics, Inc.

Inventor： Travis T. Wager ,  Zhiping Weng ,  Hualin Simon Xi

IPC: C07D403/14 ,  A61K31/4985 ,  A61K31/501 ,  A61K31/5025 ,  A61K31/506 ,  C07D405/14 ,  C07D409/14 ,  C07D413/14 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04

Abstract: The present disclosure relates to compounds of Formula (I); and to their prodrugs, pharmaceutically acceptable salts, pharmaceutical compositions, methods of use, and methods for their preparation. The compounds of the present disclosure may act as small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded genes, and methods of use of the compounds for modulating splicing and treating related diseases and conditions. The compounds disclosed herein may possess activity toward various genetic pathways and are accordingly useful in methods of treatment of the human or animal body.

公开(公告)号：US20250026753A1

公开(公告)日：2025-01-23

申请号：US18708108

申请日：2022-11-11

Applicant: Rgenta Therapeutics, Inc.

Inventor： Travis T. Wager ,  Zhiping Weng ,  Hualin Simon Xi

IPC: C07D471/04 ,  A61K31/53 ,  A61K31/551 ,  A61P35/00 ,  C07D519/00

Abstract: The present disclosure relates to compounds of Formula (I) or (II) or subformulas thereof: (I) and do their prodrugs, pharmaceutically acceptable salts, pharmaceutical compositions, methods of use, and methods for their preparation. The compounds of the present disclosure may act as small molecule splicing modulator that modulate splicing of mRNA, such as pre-mRNA, encoded genes, and methods of use of the compounds for modulating splicing and treating related diseases and conditions. The compounds disclosed herein may possess activity toward various genetic pathways and are accordingly useful in methods of treatment of the human or animal body.