公开(公告)号：US20250026736A1

公开(公告)日：2025-01-23

申请号：US18823254

申请日：2024-09-03

Applicant: NOVARTIS AG

Inventor： Simone BONAZZI ,  Artiom CERNIJENKO ,  Jennifer Stroka COBB ,  Janetta DEWHURST ,  John Ryan KERRIGAN ,  Gary O'BRIEN ,  MooJe SUNG ,  Noel Marie-France THOMSEN ,  Pamela TING

IPC: C07D401/14 ,  A61K31/4545 ,  A61K31/55 ,  A61K45/06 ,  C07D405/14

Abstract: The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.