公开(公告)号：US20190231760A1

公开(公告)日：2019-08-01

申请号：US16289612

申请日：2019-02-28

Applicant: NOVARTIS AG

Inventor： Nicole BUSCHMANN ,  Robin Alec FAIRHURST ,  Pascal FURET ,  Diana GRAUS PORTA ,  Carolina HAEFLIGER ,  Bo HAN ,  Thomas KNOPFEL ,  Catherine LEBLANC ,  Lv LIAO ,  Robert MAH ,  Masato MURAKAMI ,  Pierre NIMSGERN ,  Michael PALMER ,  Dale PORTER ,  Sebastien RIPOCHE ,  Can WANG ,  Youzhen WANG ,  Andreas WEISS ,  Jing XIONG ,  Xianglin ZHAO

IPC: A61K31/4375 ,  A61P35/00 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/55

Abstract: The present invention relates to therapeutic uses of compounds of formula (I) or a pharmaceutically acceptable salt thereof

公开(公告)号：US20190076412A1

公开(公告)日：2019-03-14

申请号：US16190087

申请日：2018-11-13

Applicant: NOVARTIS AG

Inventor： Nicole BUSCHMANN ,  Robin Alec FAIRHURST ,  Pascal FURET ,  Thomas KNÖPFEL ,  Catherine LEBLANC ,  Lv LIAO ,  Robert MAH ,  Pierre NIMSGERN ,  Sebastien RIPOCHE ,  Jing XIONG ,  Bo HAN ,  Can WANG ,  Xianglin ZHAO

IPC: A61K31/4375 ,  A61K31/4545 ,  A61P35/00 ,  A61K31/55 ,  A61K31/496

Abstract: The present invention relates to therapeutic uses of compounds of formula (1) or a pharmaceutically acceptable salt thereof.

公开(公告)号：US20180185341A1

公开(公告)日：2018-07-05

申请号：US15516443

申请日：2015-10-02

Applicant: Novartis AG

Inventor： Nicole BUSCHMANN ,  Robin Alec FAIRHURST ,  Pascal FURET ,  Diana GRAUS PORTA ,  Carolina HAEFLIGER ,  Bo HAN ,  Thomas KNÖPFEL ,  Catherine LEBLANC ,  Lv LIAO ,  Robert MAH ,  Masato MURAKAMI ,  Pierre NIMSGERN ,  Michael PALMER ,  Dale PORTER ,  Sebastien RIPOCHE ,  Can WANG ,  Youzhen WANG ,  Andreas WEISS ,  Jing XIONG ,  Xianglin ZHAO

IPC: A61K31/4375 ,  A61K31/496 ,  A61P35/00

CPC classification number: A61K31/4375 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/55 ,  A61P35/00

Abstract: The present invention relates to therapeutic uses of compounds of formula (I) or a pharmaceutically acceptable salt thereof

公开(公告)号：US20230348852A1

公开(公告)日：2023-11-02

申请号：US17997223

申请日：2021-04-26

Applicant: NOVARTIS AG ,  INTELLIA THERAPEUTICS, INC.

Inventor： Frada BERENSHTEYN ,  Bo HAN ,  Xueshi HAO ,  Jessica HEYDER ,  Timothy Z. HOFFMAN ,  Qihui JIN ,  Arnaud LACOSTE ,  Jun LIU ,  Yahu LIU ,  Tingting MO ,  Bradley Andrew MURRAY ,  Daniel Joseph O’CONNELL ,  Jianfeng PAN ,  Yun Feng XIE ,  Shanshan YAN ,  Yefen ZOU

IPC: A61P27/02 ,  C12N5/079 ,  C12N9/22

CPC classification number: C12N5/0621 ,  A61P27/02 ,  C12N9/22 ,  C12N2310/20 ,  C12N2501/999

Abstract: The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy. The invention further provides methods of generating an expanded population of genetically modified ocular cells, for example limbal stem cells (LSCs) or corneal endothelial cells (CECs), wherein the cells are expanded involving the use of a LATS inhibitor and the expression of B2M in the cells has been reduced or eliminated. The present invention also provides cell populations, preparations, uses and methods of therapy comprising said cells.