公开(公告)号：US20240358689A1

公开(公告)日：2024-10-31

申请号：US18685281

申请日：2022-08-23

申请人： Arena Pharmaceuticals, Inc.

发明人： John William Adams

IPC分类号： A61K31/438 ,  A61K31/4709 ,  A61K31/5383

CPC分类号： A61K31/438 ,  A61K31/4709 ,  A61K31/5383

摘要： The present invention relates to methods of treating renal cystic disease and/or cardiorenal syndrome by administrating certain compounds that modulate the activity of the beta-3 adrenergic receptor.

公开(公告)号：US20240327411A1

公开(公告)日：2024-10-03

申请号：US18471912

申请日：2023-09-21

申请人： The Board of Regents of The University of Texas System

发明人： Jia Zhou ,  Wenbo Zhang ,  Hua Liu

IPC分类号： C07D487/04 ,  A61K31/4985 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/541 ,  A61K31/551 ,  C07D498/04 ,  C07D519/00

CPC分类号： C07D487/04 ,  A61K31/4985 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/541 ,  A61K31/551 ,  C07D498/04 ,  C07D519/00

摘要： The present invention relates to novel small molecule activators of Sirt6, for example, compounds of the general Formula (I), their methods and use for the treatment of various human diseases such as cancer, inflammatory diseases, neurodegenerative diseases, and infectious diseases:

公开(公告)号：US20240317721A1

公开(公告)日：2024-09-26

申请号：US18571588

申请日：2022-06-22

申请人： NOVARTIS AG

发明人： Claudio BOMIO-CONFAGLIA ,  Saskia Maria BRACHMANN ,  Simona COTESTA ,  Marc GERSPACHER ,  Catherine LEBLANC ,  Fabio LIMA ,  Edwige Liliane Jeanne LORTHIOIS ,  Rainer MACHAUER ,  Robert MAH ,  Sophie RACINE ,  Pascal RIGOLLIER ,  Stefan STUTZ ,  Andrea VAUPEL ,  Nicolas WARIN ,  Rainer WILCKEN ,  Frédéric ZECRI

IPC分类号： C07D403/14 ,  A61K31/416 ,  A61K31/4162 ,  A61K31/437 ,  A61K31/438 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/496 ,  A61K31/4985 ,  A61K31/499 ,  A61K31/4995 ,  A61K31/501 ,  A61K31/5025 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/5386 ,  A61K31/541 ,  A61K31/547 ,  A61K31/55 ,  A61K31/551 ,  A61K31/553 ,  A61K31/675 ,  C07D401/14 ,  C07D405/14 ,  C07D413/14 ,  C07D417/14 ,  C07D471/04 ,  C07D471/10 ,  C07D487/04 ,  C07D487/08 ,  C07D491/048 ,  C07D491/107 ,  C07D495/10 ,  C07D498/04 ,  C07D498/08 ,  C07D498/10 ,  C07D513/08 ,  C07F9/6584

CPC分类号： C07D403/14 ,  A61K31/416 ,  A61K31/4162 ,  A61K31/437 ,  A61K31/438 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/496 ,  A61K31/4985 ,  A61K31/499 ,  A61K31/4995 ,  A61K31/501 ,  A61K31/5025 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/5386 ,  A61K31/541 ,  A61K31/547 ,  A61K31/55 ,  A61K31/551 ,  A61K31/553 ,  A61K31/675 ,  C07D401/14 ,  C07D405/14 ,  C07D413/14 ,  C07D417/14 ,  C07D471/04 ,  C07D471/10 ,  C07D487/04 ,  C07D487/08 ,  C07D491/048 ,  C07D491/107 ,  C07D495/10 ,  C07D498/04 ,  C07D498/08 ,  C07D498/10 ,  C07D513/08 ,  C07F9/6584

摘要： The present invention provides a compound of formula (I), or a pharmaceutically acceptable salt thereof, and the therapeutic uses of said compound. The present invention further provides a pharmaceutical composition comprising said compound.

公开(公告)号：US20240301372A1

公开(公告)日：2024-09-12

申请号：US18262674

申请日：2022-01-25

申请人： FRED HUTCHINSON CANCER CENTER

发明人： Keith R. Jerome ,  Barry L. Stoddard

IPC分类号： C12N9/22 ,  A61K9/00 ,  A61K31/437 ,  A61K31/517 ,  A61K31/5383 ,  A61K31/5517 ,  A61K38/00 ,  A61P31/22 ,  C12N15/86

CPC分类号： C12N9/22 ,  A61K9/0019 ,  A61K31/437 ,  A61K31/517 ,  A61K31/5383 ,  A61K31/5517 ,  A61P31/22 ,  C12N15/86 ,  A61K38/00 ,  C12N2750/14143

摘要： Embodiments of the present disclosure are directed to methods and compositions for reducing or eliminating latent HSV-1 reactivation in a cell. In some embodiments, the method comprises delivering to an HSV-1-infected cell one or more self-complementary adeno-associated viruses (scAAV) comprising one or more sequences encoding one or more HSV-1-specific meganucleases. In some embodiments, the composition comprises one or more self-complementary adeno-associated viruses (scAAV) comprising one or more sequences encoding one or more HSV-1-specific meganucleases.

公开(公告)号：US20240270756A1

公开(公告)日：2024-08-15

申请号：US18416152

申请日：2024-01-18

申请人： Ascletis BioScience Co., Ltd.

发明人： Jinzi Jason WU ,  Bin LIANG

IPC分类号： C07D498/14 ,  A61K31/4985 ,  A61K31/5383

CPC分类号： C07D498/14 ,  A61K31/4985 ,  A61K31/5383

摘要： Disclosed are a compound of Formula I, stereoisomers, pharmaceutically acceptable salts and deuterated compounds thereof and a method for treating, inhibiting and/or preventing a disease or disorder in a subject in need thereof:




wherein A, R, G, Y and n are defined as in the present disclosure.

公开(公告)号：US20240262806A1

公开(公告)日：2024-08-08

申请号：US18256099

申请日：2022-08-24

申请人： PTC THERAPEUTICS, INC.

发明人： XIAOYAN ZHANG ,  TIANYI ZHENG ,  SCOTT j BARRAZA ,  LAUREN BEJCEK ,  BRADLEY B GILBERT ,  HUA GONG ,  HANDOKO HANDOKO ,  SEYEDMORTEZA HOSSEYNI ,  EDUARDO HUARTE ,  WOOHYUNG JEON ,  JING LI ,  YAO LIU ,  KYLE NIEDERER ,  MEENU PILLAI ,  ERICA N PARKER ,  ETTORE RASTELLI ,  NADIYA SYDORENKO ,  ANTHONY TURPOFF ,  MATTHEW G WOLL ,  NANJING ZHANG ,  YAN ZHANG ,  RAUFUL ALAM

IPC分类号： C07D401/12 ,  A61K31/395 ,  A61K31/502 ,  A61K31/5025 ,  A61K31/53 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/55 ,  A61K31/551 ,  A61K31/553 ,  C07D237/34 ,  C07D401/14 ,  C07D403/12 ,  C07D405/14 ,  C07D413/12 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D491/048 ,  C07D495/04 ,  C07D498/04 ,  C07D519/00

CPC分类号： C07D401/12 ,  A61K31/502 ,  A61K31/5025 ,  A61K31/53 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/55 ,  A61K31/551 ,  A61K31/553 ,  A61K31/5545 ,  C07D237/34 ,  C07D401/14 ,  C07D403/12 ,  C07D405/14 ,  C07D413/12 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D491/048 ,  C07D495/04 ,  C07D498/04 ,  C07D519/00

摘要： The present invention relates to novel compounds of Formulae I-XI: wherein each A, A′, Q, Q′, W, Rw, Y, and Z, and -- are as defined herein, which inhibit NOD-like receptor protein 3 (NLRP3) inflammasome activity. The invention further relates to the processes for their preparation, pharmaceutical compositions and medicaments containing them, and their use in the treatment of diseases and disorders mediated by NLRP3.

公开(公告)号：US12016853B2

公开(公告)日：2024-06-25

申请号：US17228590

申请日：2021-04-12

申请人： Belite Bio, Inc ,  THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK

发明人： Yu-Hsin Tom Lin ,  Cheng-Chi Irene Wang ,  Konstantin Petrukhin

IPC分类号： A61K31/4545 ,  A61K9/00 ,  A61K31/4985 ,  A61K31/519 ,  A61K31/5365 ,  A61K31/5383 ,  A61P3/04 ,  A61P3/10

CPC分类号： A61K31/4545 ,  A61K9/0019 ,  A61K9/0053 ,  A61K31/4985 ,  A61K31/519 ,  A61K31/5365 ,  A61K31/5383 ,  A61P3/04 ,  A61P3/10

摘要： Provided herein are heterocyclic derivative compounds and pharmaceutical compositions comprising said compounds that are useful for the treatment of retinal binding protein (RBP4) related diseases, such as obesity and the like.

公开(公告)号：US20240199630A1

公开(公告)日：2024-06-20

申请号：US18551912

申请日：2022-03-23

申请人： FUNDACIÓN DEL SECTOR PÚBLICO ESTATAL CENTRO NACIONAL DE INVESTIGACIONES ONCOLÓGICAS

发明人： Joaquín PASTOR FERNÁNDEZ ,  Sonia MARTÍNEZ GONZÁLEZ ,  Carmen BLANCO APARICIO ,  Ana Belén GARCÍA GARCÍA ,  Sonsoles RODRÍGUEZ ARÍSTEGUI ,  Cristina Ana GÓMEZ DE LA OLIVA ,  María Isabel ALBARRÁN SANTIÑO ,  Antonio CEBRIÁ GÓMEZ ,  Marcos MALUMBRES MARTÍNEZ

IPC分类号： C07D491/147 ,  A61K31/145 ,  A61K31/15 ,  A61K31/4375 ,  A61K31/4439 ,  A61K31/50 ,  A61K31/502 ,  A61K31/5025 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/55 ,  A61K31/566 ,  A61K31/635 ,  C07D498/14

CPC分类号： C07D491/147 ,  A61K31/145 ,  A61K31/15 ,  A61K31/4375 ,  A61K31/4439 ,  A61K31/50 ,  A61K31/502 ,  A61K31/5025 ,  A61K31/5377 ,  A61K31/5383 ,  A61K31/55 ,  A61K31/566 ,  A61K31/635 ,  C07D498/14

摘要： The present invention relates to a group of compounds with a tricyclic core based on imidazo[1,2-b]pyridazine of formula (I):




which are inhibitors of HASPIN, whose activity is required for the proliferation of certain tumoral cells, so the compounds of the invention are useful for the prevention and/or treatment of cancer, alone or in combination with chemotherapeutic agents.

公开(公告)号：US20240165123A1

公开(公告)日：2024-05-23

申请号：US18280868

申请日：2022-03-09

申请人： CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd. ,  Hangzhou Innogate Pharma Co., Ltd ,  CSPC Ouyi Pharmaceutical Co., Ltd

发明人： Hanyu YANG ,  Hancheng ZHANG ,  Xibao LIU ,  Congcong CAI ,  Ning QIN ,  Mo DAN ,  Lu LYU ,  Dandan ZHANG ,  Jieru LIU

IPC分类号： A61K31/5383 ,  A61P17/06 ,  A61P37/06

CPC分类号： A61K31/5383 ,  A61P17/06 ,  A61P37/06

摘要： The present application provides use of Compound (I), an optical isomer thereof or a pharmaceutically acceptable salt thereof in manufacture of a medicament useful in treating a disease related to high expression or abnormal activation of JAK and SYK kinases, comprising an autoimmune disease, such as an immune-mediated skin disease, in particular psoriasis, atopic dermatitis and SLE. Compound (I), an optical isomer thereof or a pharmaceutically acceptable salt thereof according to the present invention can improve the skin lesions in mice with psoriasis, atopic dermatitis and SLE, alleviate the injury of kidney, inhibit the expansion of immune organs, reduce the level of inflammation, inhibit the increase of SLE related antibodies and cytokines in serum, and has a certain safe therapeutic window, which has a good clinical application prospect.

公开(公告)号：US20240158398A1

公开(公告)日：2024-05-16

申请号：US18137228

申请日：2023-04-20

申请人： Blueprint Medicines Corporation

发明人： Natasja Brooijmans ,  Jason D. Brubaker ,  Paul E. Fleming ,  Brian Lewis Hodous ,  Joseph L. Kim ,  Brett D. Williams ,  Douglas Wilson ,  Kevin J. Wilson ,  Mark Cronin

IPC分类号： C07D487/04 ,  A61K31/5025 ,  A61K31/5377 ,  A61K31/5383 ,  A61P35/00 ,  C07D519/00

CPC分类号： C07D487/04 ,  A61K31/5025 ,  A61K31/5377 ,  A61K31/5383 ,  A61P35/00 ,  C07D519/00

摘要： Described herein are compounds that inhibit ALK2 and its mutants, pharmaceutical compositions including such compounds, and methods of using such compounds and compositions.