公开(公告)号：US12172984B2

公开(公告)日：2024-12-24

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

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：US11787785B2

公开(公告)日：2023-10-17

申请号：US17693759

申请日：2022-03-14

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 Y F 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：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：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：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.