公开(公告)号：US20230067476A1

公开(公告)日：2023-03-02

申请号：US17520857

申请日：2021-11-08

Applicant: Novartis AG

Inventor： Claire ADCOCK ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Philip LAM ,  Kathryn Taylor LINKENS ,  Hasnain Ahmed MALIK ,  Noel Marie-France THOMSEN ,  Michael Scott VISSER

IPC: C07D401/04 ,  C07D407/14

Abstract: The present disclosure provides a compound of Formula (I′): or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof, wherein Rx, X1, X2, and R1 are as defined herein, and methods of making and using same.

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：US20220144798A1

公开(公告)日：2022-05-12

申请号：US17430478

申请日：2020-02-13

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Philip LAM ,  Noel Marie-France THOMSEN

IPC: C07D401/04 ,  C07D401/14 ,  C07D471/04

Abstract: The present disclosure provides a compound of Formula (I) or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof, wherein R1, R2, Rx, X1, X2, X3, n, n1, and q are as defined herein, and methods of making and using same.

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：US20200017461A1

公开(公告)日：2020-01-16

申请号：US16504375

申请日：2019-07-08

Applicant: Novartis AG

Inventor： Claire ADCOCK ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Philip LAM ,  Kathryn Taylor LINKENS ,  Hasnain Ahmed MALIK ,  Noel Marie-France THOMSEN ,  Michael Scott VISSER

IPC: C07D401/04 ,  C07D407/14

Abstract: The present disclosure provides a compound of Formula (I′): or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof, wherein Rx, X1, X2, and R1 are as defined herein, and methods of making and using same.