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：US20220144807A1

公开(公告)日：2022-05-12

申请号：US17430473

申请日：2020-02-13

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Michael Scott VISSER

IPC: C07D401/14 ,  A61K31/4545 ,  A61K31/506 ,  A61K31/501 ,  A61K31/497 ,  A61P35/00

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, R3, Rx, and n are as defined herein, and methods of making and using same.

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：US20190062309A1

公开(公告)日：2019-02-28

申请号：US16108713

申请日：2018-08-22

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Aleem FAZAL ,  Ritesh Bhanudasji TICHKULE ,  Michael Scott VISSER

IPC: C07D401/14 ,  A61P35/00

CPC classification number: C07D401/14 ,  A61P35/00 ,  C07D401/04 ,  C07D407/14 ,  C07D409/14 ,  C07D413/14 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D487/08 ,  C07D495/04 ,  C07D513/04

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, n, n1, and q are as defined herein, and methods of making and using same.

公开(公告)号：US20240245670A1

公开(公告)日：2024-07-25

申请号：US17415923

申请日：2019-12-19

Applicant: Novartis AG

Inventor： Simone BONAZZI ,  Adam Crystal ,  John Scott Cameron ,  Eva Marie Genevieve D'Hennezel ,  Glenn Dranoff ,  Ry Roger Forseth ,  Dominik Johannes Hainzl ,  Jacqueline Kinyamu-Akunda ,  Guiqing Liang ,  Lilly Mary Petruzzelli

IPC: A61K31/4545 ,  A61K9/00 ,  A61K31/55 ,  A61P35/00

CPC classification number: A61K31/4545 ,  A61K9/0053 ,  A61K31/55 ,  A61P35/00

Abstract: The present disclosure relates to dosing regimens, formulations, and combinations comprising 3-(1-oxoisoindolin-2-yl)piperidine-2,6-dione compounds or pharmaceutical compositions comprising the same; and methods of using such combinations and compositions in the treatment or prevention IKAROS Family Zinc Finger 2 (IKZF2)-dependent diseases or disorders or where reduction of IKZF2 or IKZF4 protein levels can ameliorate a disease, for example, the treatment of cancers.

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：US20230271940A1

公开(公告)日：2023-08-31

申请号：US18006894

申请日：2021-07-29

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO

IPC: C07D401/14 ,  C07D405/14 ,  C07D471/04

CPC classification number: C07D401/14 ,  C07D405/14 ,  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 Rx and X1 are as defined herein, and methods of making and using same.

公开(公告)号：US20200231569A1

公开(公告)日：2020-07-23

申请号：US16835938

申请日：2020-03-31

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Aleem FAZAL ,  Ritesh Bhanudasji TICHKULE ,  Michael Scott VISSER

IPC: C07D401/14 ,  C07D401/04 ,  C07D407/14 ,  C07D409/14 ,  C07D413/14 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D487/08 ,  C07D495/04 ,  C07D513/04 ,  A61P35/00

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, n, n1, and q are as defined herein, and methods of making and using same.

公开(公告)号：US20200016143A1

公开(公告)日：2020-01-16

申请号：US16504376

申请日：2019-07-08

Applicant: Novartis AG

Inventor： Rohan Eric John BECKWITH ,  Simone BONAZZI ,  Artiom CERNIJENKO ,  Fupeng MA ,  Nathaniel F. WARE

IPC: A61K31/454 ,  C07D401/14 ,  C07D401/04 ,  A61K31/4545 ,  A61P35/00 ,  A61K31/498

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