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

IPC: A61K35/14 ,  A61K35/12 ,  A61P7/00 ,  C12N5/071 ,  C12N9/22 ,  C12N15/113 ,  C12N15/52

Abstract: The present invention is directed to compositions and methods for the treatment of hemoglobinopathies.

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.

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.

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.

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.

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.

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.