公开(公告)号：US12172984B2

公开(公告)日：2024-12-24

申请号：US18460428

申请日：2023-09-01

Applicant: Novartis AG

Inventor： Simone Bonazzi ,  Artiom Cernijenko ,  Jennifer Stroka Cobb ,  Natalie Alysia Dales ,  Janetta Dewhurst ,  Matthew James Hesse ,  Rama Jain ,  John Ryan Kerrigan ,  Hasnain Ahmed Malik ,  James R. Manning ,  Gary O'Brien ,  Andrew W. Patterson ,  Noel Marie-France Thomsen ,  Pamela Yf Ting

IPC: C07D403/14 ,  A61P7/00 ,  C07D405/14 ,  C07D417/14

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.

公开(公告)号：US12252482B2

公开(公告)日：2025-03-18

申请号：US18826887

申请日：2024-09-06

Applicant: Novartis AG

Inventor： Simone Bonazzi ,  Jennifer Stroka Cobb ,  Natalie Alysia Dales ,  Matthew James Hesse ,  Rama Jain ,  John Ryan Kerrigan ,  Hasnain Ahmed Malik ,  James R Manning ,  Pamela Yf Ting

IPC: C07D403/14 ,  A61P7/00 ,  C07D405/14 ,  C07D417/14

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.