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公开(公告)号:US20230279394A1
公开(公告)日:2023-09-07
申请号:US17786347
申请日:2020-12-16
Applicant: Novartis AG
Inventor: Muluken BELEW , Simone BONAZZI , James BRADNER , Artiom CERNIJENKO , Jennifer Stroka COBB , Natalie DALES , John Ryan KERRIGAN , Philip LAM , Hasnain Ahmed MALIK , Carsten RUSS , Frederic SIGOILLOT , Susan C. STEVENSON , Noel Marie-France THOMSEN , Pamela TING
IPC: C12N15/113 , C12N15/86 , C12N5/07 , A61P7/00
CPC classification number: C12N15/113 , A61P7/00 , C12N5/06 , C12N15/86 , C12N2310/20
Abstract: The present invention is directed to compositions and methods for the treatment of hemoglobinopathies.
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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.
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公开(公告)号:US20220402904A1
公开(公告)日:2022-12-22
申请号:US17124127
申请日:2020-12-16
Applicant: NOVARTIS AG
Inventor: Simone BONAZZI , Artiom CERNIJENKO , Jennifer Stroka COBB , Natalie DALES , John Ryan KERRIGAN , Philip LAM , Hasnain Ahmed MALIK , Gary O'BRIEN , Andrew W. PATTERSON , Noel Marie-France THOMSEN , Pamela TING
IPC: C07D413/14 , C07D401/14 , C07D471/04 , C07D417/14 , A61K45/06
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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