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公开(公告)号：US20250002479A1
公开(公告)日：2025-01-02
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.
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公开(公告)号：US20240158374A1
公开(公告)日：2024-05-16
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
CPC classification number: 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.
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公开(公告)号：US20250127809A1
公开(公告)日：2025-04-24
申请号：US18569029
申请日：2022-06-22
Applicant: Novartis AG
Inventor： Muluken Shambel BELEW , Jennifer Stroka COBB , Carsten RUSS , Frederic SIGOILLOT , Susan C. STEVENSON , Pamela YF TING
Abstract: The present invention is directed to compositions and methods for the treatment of hemoglobinopathies.
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