公开(公告)号：US20230398224A1

公开(公告)日：2023-12-14

申请号：US17760081

申请日：2021-02-04

申请人： Novartis AG

发明人： Martin Allan ,  Jeffrey T. BAGDANOFF ,  David Weninger Barnes ,  Kevin Clairmont ,  Thomas Smith ,  Shuangxi Wang

IPC分类号： A61K47/54 ,  C07K7/64

CPC分类号： A61K47/549 ,  C07K7/64

摘要： The present invention is directed to the bifunctional compounds and the use of such bifunctional compounds to lower plasma levels of extracellular target molecules by lysosomal degradation. Such bifunctional compounds have a cell surface receptor ligand covalently linked to a ligand that is capable of binding to an extracellular target molecule (such as a ligand for a growth factor, a cytokine, a chemokine, a hormone, a neurotransmitter, a capsid, a soluble receptor, an extracellular secreted protein, an antibody, a lipoprotein, an exosome, a virus, a cell, or a plasma membrane protein), where the cell surface receptor is associated with receptor mediated endocytosis, including asialoglycoprotein receptor (ASGPR) mediated lysosomal degradation and mannose-6-phosphate (M6PR) mediated lysosomal degradation. Pharmaceutical compositions comprising such bifunctional compounds and methods of treating a disease or disorder mediated by an extracellular molecule using such bifunctional compounds are also provided herein.

公开(公告)号：US20180369386A1

公开(公告)日：2018-12-27

申请号：US16115883

申请日：2018-08-29

申请人： Novartis AG

发明人： Jeremy Lee Baryza ,  Rohan Eric John Beckwith ,  Keith Bowman ,  Crystal Byers ,  Tanzina Fazal ,  Gabriel Grant Gamber ,  Cameron Chuck-Munn Lee ,  Ritesh Bhanudasji Tichkule ,  Chandra Vargeese ,  Shuangxi Wang ,  Laura West ,  Thomas Zabawa ,  Junping Zhao

IPC分类号： A61K47/18 ,  C07C229/14 ,  A61K9/127 ,  A61K9/51 ,  C12N15/113 ,  C07D317/28 ,  C07D295/096 ,  C07D213/79 ,  C07D213/69 ,  C07D205/04 ,  C07C235/42 ,  A61K9/00 ,  C07C229/12 ,  C07C219/06 ,  C07C217/58 ,  A61K47/22 ,  A61K38/22 ,  A61K31/713 ,  A61K48/00

摘要： This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

公开(公告)号：US12129246B1

公开(公告)日：2024-10-29

申请号：US18589841

申请日：2024-02-28

申请人： Novartis AG

发明人： Daniela Angst ,  Philippe Bolduc ,  Matthew William Carson ,  Atwood Kim Cheung ,  Véronique Darsigny ,  Xiang Gao ,  Viktor Hornak ,  Keith Jendza ,  Rajesh Karki ,  Ajay Kumar Lal ,  Gang Liu ,  Justin Yik Ching Mao ,  Jeffrey M. McKenna ,  Erik Meredith ,  Muneto Mogi ,  Vivek Rauniyar ,  Liansheng Su ,  Ritesh Tichkule ,  Shuangxi Wang ,  Chun Zhang ,  Liang Zhao ,  Rui Zheng

IPC分类号： C07D403/14 ,  A61K31/517 ,  A61K31/55 ,  A61K45/06 ,  C07D239/95 ,  C07D401/12 ,  C07D401/14

CPC分类号： C07D403/14 ,  A61K31/517 ,  A61K31/55 ,  A61K45/06 ,  C07D239/95 ,  C07D401/12 ,  C07D401/14

摘要： The disclosure relates to a compound of Formula (I):




or a pharmaceutically acceptable salt thereof wherein A, Ra to Rd, and R4 to R7, are as described herein, as well as compositions and methods of using such compounds.

公开(公告)号：US10792361B2

公开(公告)日：2020-10-06

申请号：US16115883

申请日：2018-08-29

申请人： Novartis AG

发明人： Jeremy Lee Baryza ,  Rohan Eric John Beckwith ,  Keith Bowman ,  Crystal Byers ,  Tanzina Fazal ,  Gabriel Grant Gamber ,  Cameron Chuck-Munn Lee ,  Ritesh Bhanudasji Tichkule ,  Chandra Vargeese ,  Shuangxi Wang ,  Laura West ,  Thomas Zabawa ,  Junping Zhao

IPC分类号： A61K47/18 ,  A61K9/127 ,  A61K9/51 ,  A61K31/713 ,  C07C217/58 ,  C07C219/06 ,  C07C229/12 ,  C07D317/28 ,  C07D295/096 ,  A61K9/00 ,  A61K38/22 ,  A61K47/22 ,  C07C229/14 ,  C07C235/42 ,  C07D205/04 ,  C07D213/69 ,  C07D213/79 ,  C12N15/113 ,  A61K48/00

摘要： This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

公开(公告)号：US10729775B2

公开(公告)日：2020-08-04

申请号：US16021193

申请日：2018-06-28

申请人： Novartis AG

发明人： Jeremy Lee Baryza ,  Rohan Eric John Beckwith ,  Keith Bowman ,  Crystal Byers ,  Tanzina Fazal ,  Gabriel Grant Gamber ,  Cameron Chuck-munn Lee ,  Ritesh Bhanudasji Tichkule ,  Chandra Vargeese ,  Shuangxi Wang ,  Laura Ellen West ,  Thomas Zabawa ,  Junping Zhao

IPC分类号： A61K47/18 ,  C12N15/113 ,  A61K47/22 ,  C07D213/79 ,  C07D317/24 ,  C07D317/28 ,  C07D205/04 ,  C07D295/096 ,  C07D213/69 ,  A61K9/127 ,  C07C217/58 ,  C07C217/60 ,  C07C217/62 ,  C07C219/22 ,  C07C219/28 ,  C07C229/12 ,  C07C235/46 ,  C07C237/08

摘要： This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

公开(公告)号：US10124065B2

公开(公告)日：2018-11-13

申请号：US14779939

申请日：2014-03-06

申请人： Novartis AG

发明人： Jeremy Lee Baryza ,  Rohan Eric John Beckwith ,  Keith Bowman ,  Crystal Byers ,  Tanzina Fazal ,  Gabriel Grant Gamber ,  Cameron Chuck-Munn Lee ,  Ritesh Bhanudasji Tichkule ,  Chandra Vargeese ,  Shuangxi Wang ,  Laura West ,  Thomas Zabawa ,  Junping Zhao

IPC分类号： C07C211/00 ,  A61K9/00 ,  C07C217/00 ,  A61K47/22 ,  A61K47/18 ,  C07C217/58 ,  C07C219/06 ,  C07C229/12 ,  C07D317/28 ,  C07D295/096 ,  A61K9/127 ,  A61K9/51 ,  A61K31/713 ,  A61K38/22 ,  C07C229/14 ,  C07C235/42 ,  C07D205/04 ,  C07D213/69 ,  C07D213/79 ,  C12N15/113 ,  A61K48/00

摘要： This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

公开(公告)号：US20180296677A1

公开(公告)日：2018-10-18

申请号：US16021193

申请日：2018-06-28

申请人： Novartis AG

发明人： Jeremy Lee Baryza ,  Rohan Eric John Beckwith ,  Keith Bowman ,  Crystal Byers ,  Tanzina Fazal ,  Gabriel Grant Gamber ,  Cameron Chuck-munn Lee ,  Ritesh Bhanudasji Tichkule ,  Chandra Vargeese ,  Shuangxi Wang ,  Laura Ellen West ,  Thomas Zabawa ,  Junping Zhao

IPC分类号： A61K47/18 ,  C07C235/46 ,  A61K9/127 ,  C12N15/113 ,  C07D317/28 ,  C07D317/24 ,  C07D295/096 ,  C07D213/79 ,  C07D213/69 ,  C07D205/04 ,  C07C237/08 ,  C07C229/12 ,  C07C219/28 ,  C07C219/22 ,  C07C217/62 ,  C07C217/60 ,  C07C217/58 ,  A61K47/22

摘要： This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.