公开(公告)号：US20230019617A1

公开(公告)日：2023-01-19

申请号：US17829933

申请日：2022-06-01

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 ,  A61K45/06 ,  C07D405/14 ,  A61K31/55

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：US12281125B2

公开(公告)日：2025-04-22

申请号：US17414696

申请日：2019-12-17

Applicant: NOVARTIS AG

Inventor： Simone Bonazzi ,  Michael Connolly ,  David Jonathan Glass ,  Manuel Mihalic ,  Andrew W. Patterson ,  Silvio Roggo ,  Tea Shavlakadze ,  Patrik Zueger

IPC: C07D498/18 ,  A61K31/436 ,  A61K45/06

Abstract: The disclosure provides 32-deoxo-rapamycin derivatives of Formula (I), or a pharmaceutically acceptable salt thereof, pharmaceutical compositions and pharmaceutical combinations comprising a compound of Formula (I), and methods of making same. Also provided herein are methods of using a compound of Formula (I) or a pharmaceutically acceptable salt thereof for treating the diseases and disorders described herein.

公开(公告)号：US12252482B2

公开(公告)日：2025-03-18

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：US20220202787A1

公开(公告)日：2022-06-30

申请号：US17442733

申请日：2020-03-25

Applicant: NOVARTIS AG

Inventor： Simone Bonazzi ,  Michael Connolly ,  David Jonathan Glass ,  Andrew W. Patterson ,  Tea Shavlakadze

IPC: A61K31/439 ,  A61K45/06 ,  C07D498/18

Abstract: Described herein are compounds of Formula (I) that are inhibitors of mTORC1, pharmaceutical compositions including such compounds, and methods of using such compounds and compositions.

公开(公告)号：US12103919B2

公开(公告)日：2024-10-01

申请号：US17829933

申请日：2022-06-01

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

CPC classification number: 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：US11192877B2

公开(公告)日：2021-12-07

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.

公开(公告)号：US11053218B2

公开(公告)日：2021-07-06

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 ,  A61K31/4412 ,  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：US10414755B2

公开(公告)日：2019-09-17

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 ,  A61K31/4412 ,  A61P35/00 ,  C07D413/14 ,  C07D401/04 ,  C07D407/14 ,  C07D409/14 ,  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：US11566022B2

公开(公告)日：2023-01-31

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 ,  A61K45/06 ,  C07D417/14 ,  C07D471/04

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, including hemoglobinopathies, sickle cell disease and beta-thalassemia.

公开(公告)号：US20220064185A1

公开(公告)日：2022-03-03

申请号：US17414696

申请日：2019-12-17

Applicant: NOVARTIS AG

Inventor： Simone Bonazzi ,  Michael Connolly ,  David Jonathan Glass ,  Manuel Mihalic ,  Andrew W. Patterson ,  Silvio Roggo ,  Tea Shavlakadze ,  Patrik Zueger

IPC: C07D498/18 ,  A61K45/06 ,  A61K31/436

Abstract: The disclosure provides 32-deoxo-rapamycin derivatives of Formula (I), or a pharmaceutically acceptable salt thereof, pharmaceutical compositions and pharmaceutical combinations comprising a compound of Formula (I), and methods of making same. Also provided herein are methods of using a compound of Formula (I) or a pharmaceutically acceptable salt thereof for treating the diseases and disorders described herein.