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.

公开(公告)号：US20230190751A1

公开(公告)日：2023-06-22

申请号：US17820613

申请日：2022-08-18

Applicant: Novartis AG

Inventor： Dirk BEHNKE ,  Frada BERENSHTEYN ,  Xueshi HAO ,  Timothy Z. HOFFMAN ,  Qihui JIN ,  Arnaud LACOSTE ,  Cameron LEE ,  Jun LIU ,  Yahu A. LIU ,  Juergen Klaus MAIBAUM ,  Tingting MO ,  Jianfeng PAN ,  Xin QU ,  Jan TCHORZ ,  Yun Feng XIE ,  Shanshan YAN ,  Yefen ZOU

IPC: A61K31/519 ,  C07D487/04 ,  C12N5/079 ,  C07D519/00 ,  A61P17/02 ,  A61K9/00 ,  A61K31/4375 ,  A61K31/47 ,  A61K31/506

CPC classification number: A61K31/519 ,  C07D487/04 ,  C12N5/0621 ,  C07D519/00 ,  A61P17/02 ,  A61K9/0048 ,  A61K31/4375 ,  A61K31/47 ,  A61K31/506 ,  A61K35/30

Abstract: The present invention is related to 6-6 Fused Bicyclic Heteroaryl Compounds of the Formula A2 or A1 and their Use as LATS Inhibitors, or a salt, stereoisomer or pharmaceutical composition thereof; wherein the variables are as defined herein.




The present invention further relates to a method of LATS inhibition in a cell population using a compound of Formula A1, or a salt, stereoisomer or pharmaceutical composition thereof. The present invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, their use in the treatment and management of diseases or disorders.

公开(公告)号：US20220127247A1

公开(公告)日：2022-04-28

申请号：US17428353

申请日：2020-02-04

Applicant: NOVARTIS AG

Inventor： Mihai AZIMIOARA ,  Bei CHEN ,  Robert EPPLE ,  James Paul LAJINESS ,  Casey Jacob Nelson MATHISON ,  Juliet NABAKKA ,  Victor Ivanovich NIKULIN ,  Sejal PATEL ,  Dean Paul PHILLIPS ,  Paul Vincent RUCKER ,  Andrew VALIERE ,  Baogen WU ,  Shanshan YAN ,  Xuefeng ZHU

IPC: C07D401/12 ,  C07D401/14 ,  A61K45/06 ,  A61K31/444 ,  A61K31/4545 ,  A61K31/506 ,  A61K31/496 ,  C07D405/14

Abstract: The invention relates to heterocyclic compounds of the formula (I) in which all of the variables are as defined in the specification; capable of modulating the activity of CFTR. The invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, in particular to their use in the treatment and management of diseases or disorders including Cystic fibrosis and related disorders.

公开(公告)号：US20220071971A1

公开(公告)日：2022-03-10

申请号：US17415335

申请日：2019-12-18

Applicant: NOVARTIS AG

Inventor： Mihai Azimioara ,  Badry Bursulaya ,  Songchun Jiang ,  Casey Jacob Nelson MATHISON ,  Victor Ivanovich NIKULIN ,  Truc Ngoc NGUYEN ,  Barun OKRAM ,  Sejal PATEL ,  Dean Paul PHILLIPS ,  Lewis WHITEHEAD ,  Baogen WU ,  Shanshan YAN ,  Xuefeng ZHU

IPC: A61K31/439 ,  A61K45/06 ,  C07D515/18 ,  C07D513/22 ,  C07D513/18 ,  A61K31/5377

Abstract: The invention relates to heterocyclic compounds of the formula (I), in which all of the variables are as defined in the specification; capable of modulating the activity of CFTR. The invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, in particular to their use in the treatment and management of diseases or disorders including Cystic fibrosis and related disorders.

公开(公告)号：US20180344738A1

公开(公告)日：2018-12-06

申请号：US15963816

申请日：2018-04-26

Applicant: Novartis AG

Inventor： Dirk BEHNKE ,  Frada BERENSHTEYN ,  Xueshi HAO ,  Timothy HOFFMAN ,  Qihui JIN ,  Arnaud LACOSTE ,  Cameron LEE ,  Jun LIU ,  Yahu LIU ,  Juergen Klaus MAIBAUM ,  Tingting MO ,  Jianfeng PAN ,  Xin QU ,  Jan TCHORZ ,  Yun Feng XIE ,  Shanshan YAN ,  Yefen ZOU

IPC: A61K31/519 ,  A61K9/00 ,  A61K31/4375 ,  A61K31/506 ,  A61K31/47

CPC classification number: C07D519/00 ,  A61K35/30 ,  A61P17/02 ,  C07D487/04 ,  C12N5/0621

Abstract: The present invention is related to 6-6 Fused Bicyclic Heteroaryl Compounds of the Formula A2 or A1 and their Use as LATS Inhibitors, or a salt, stereoisomer or pharmaceutical composition thereof; wherein the variables are as defined herein. The present invention further relates to a method of LATS inhibition in a cell population using a compound of Formula A1, or a salt, stereoisomer or pharmaceutical composition thereof. The present invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, their use in the treatment and management of diseases or disorders.