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：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：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：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：US20190055219A1

公开(公告)日：2019-02-21

申请号：US16045993

申请日：2018-07-26

Applicant: NOVARTIS AG

Inventor： John Ryan Kerrigan ,  Natalie Dales ,  George Scott Tria ,  Daniel Palacios ,  Paul Gormisky

IPC: C07D401/14 ,  C07D405/14 ,  C07D403/12 ,  C07D471/04 ,  C07D498/04 ,  C07D403/14

Abstract: The present disclosure provides a compound of formula (I): or a pharmaceutically acceptable salt thereof, and its therapeutic uses for activating a growth factor pathway, promoting wound healing, promoting tissue repair, and treating hearing loss, skeletal muscle loss, organ degeneration, tissue damage, neurodegeneration, and muscular atrophy. The disclosure further provides pharmaceutical compositions and combinations. The present disclosure also relates to the use of such compounds for research or other non-therapeutic purposes.

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：US10654828B2

公开(公告)日：2020-05-19

申请号：US16045993

申请日：2018-07-26

Applicant: NOVARTIS AG

Inventor： John Ryan Kerrigan ,  Natalie Dales ,  George Scott Tria ,  Daniel Palacios ,  Paul Gormisky

IPC: C07D401/14 ,  C07D405/14 ,  C07D403/14 ,  C07D471/04 ,  C07D487/04 ,  C07D403/04 ,  A61P25/28 ,  C07D471/08 ,  C07D491/04 ,  A61P21/00 ,  A61P27/16 ,  C07D403/12 ,  C07D498/04

Abstract: The present disclosure provides a compound of formula (I): or a pharmaceutically acceptable salt thereof, and its therapeutic uses for activating a growth factor pathway, promoting wound healing, promoting tissue repair, and treating hearing loss, skeletal muscle loss, organ degeneration, tissue damage, neurodegeneration, and muscular atrophy. The disclosure further provides pharmaceutical compositions and combinations. The present disclosure also relates to the use of such compounds for research or other non-therapeutic purposes.