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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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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: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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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:US20220089639A1
公开(公告)日:2022-03-24
申请号:US17228284
申请日:2021-04-12
Applicant: Novartis AG
Inventor: Aimee Richardson USERA , Zachary Hayes ROBINSON , Jennifer Stroka COBB
IPC: C07K5/103 , C07K5/083 , C07K5/062 , A61K49/00 , C07C271/22 , C07C317/28 , C07K1/13 , C07K5/072 , A61K47/64 , C07D229/02 , C07D257/08 , C07D209/42 , C07K7/02
Abstract: The present invention relates to methods and reagents for use in site-selective modification of proteins having lysine residues with functionalized peptides using a chemoenzymatic microbial transglutaminase-mediated reaction. The functionalized proteins may be used for study or therapeutic uses.
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