公开(公告)号：EP4429714A2

公开(公告)日：2024-09-18

申请号：EP22893527.6

申请日：2022-11-08

申请人： Stoke Therapeutics, Inc.

发明人： AZNAREZ, Isabel ,  LIM, Kian Huat ,  KACH, Jacob

IPC分类号： A61K48/00 ,  A61K31/7088 ,  A61K39/395 ,  A61P27/02

CPC分类号： A61K31/712 ,  C12Y306/05005 ,  C12N15/1137 ,  C12N2310/1120130101 ,  C12N2310/31520130101 ,  C12N2320/1020130101 ,  C12N2320/3320130101 ,  C12N2310/323120130101 ,  C12N2310/334120130101 ,  C12N2310/34320130101 ,  C12N9/16

公开(公告)号：EP4119569B1

公开(公告)日：2024-07-31

申请号：EP22180697.9

申请日：2016-11-07

IPC分类号： C07H19/20 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  A61P3/06

CPC分类号： C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C07H21/00 ,  C12N2310/34120130101 ,  C12N2310/351520130101 ,  A61P3/06 ,  A61K31/712 ,  C07H19/20

公开(公告)号：EP4402260A1

公开(公告)日：2024-07-24

申请号：EP22870970.5

申请日：2022-09-16

申请人： Ionis Pharmaceuticals, Inc.

发明人： FREIER, Susan, M. ,  BUI, Huynh-Hoa

IPC分类号： C12N15/11 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/00

CPC分类号： A61K31/712 ,  C12N2310/1120130101 ,  C12N15/1137 ,  C12N2310/323120130101 ,  C12N2310/34120130101 ,  C12N2310/31520130101 ,  C12N2320/1120130101 ,  C12N2310/334120130101

公开(公告)号：EP4366785A1

公开(公告)日：2024-05-15

申请号：EP22741558.5

申请日：2022-07-04

申请人： UCL Business Ltd

发明人： ZHOU, Haiyan ,  GISSEN, Paul ,  DORESTE, Bruno

IPC分类号： A61K48/00 ,  A61P43/00

CPC分类号： A61P43/00 ,  C12N15/1137 ,  C12N2310/1120130101 ,  C12N2310/1420130101 ,  C12N2310/34120130101 ,  C12N2310/31520130101 ,  C12N2320/3120130101 ,  A61K31/122 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/711 ,  A61K31/712 ,  Y02A50/30

公开(公告)号：EP3400300A1

公开(公告)日：2018-11-14

申请号：EP17736357.9

申请日：2017-01-05

申请人： Ionis Pharmaceuticals, Inc.

发明人： ZHAO, Hien, Thuy ,  KORDASIEWICZ, Holly

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125

CPC分类号： C12N15/1137 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P25/16 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Y207/11001 ,  C12N2310/321 ,  C12N2310/3521

摘要： Provided herein are methods for decreasing LRRK2 mRNA expression. Such methods are useful to ameliorate LRRK2 associated diseases. Such LRRK2 associated diseases include Parkinson's Disease, including non-LRRK2 mediated Parkinson's Disease.

公开(公告)号：EP3393479A1

公开(公告)日：2018-10-31

申请号：EP16877042.8

申请日：2016-12-13

申请人： Sarissa Inc.

发明人： VINCENT, Mark D. ,  FERGUSON, Peter ,  KOROPATNICK, James D. ,  RYTELEWSKI, Mateusz

IPC分类号： A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61P35/00 ,  C07H21/00 ,  C12N15/113

CPC分类号： A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3521

摘要： Methods of treating cancer using antisense based therapies including antisense oligonucleotides or si RNAs directed against DNA double-strand break repair proteins such as BRCA2 or RAD51 are provided. The antisense based therapies can be used alone, in tandem or in combination with other cancer therapies, in particular with therapies that lead to DNA damage, inhibition of DNA repair or inhibition of DNA synthesis, such as radiation, platinum drugs, alkylating agents, PARP inhibitors, or inhibitors of thymidylate synthase.

公开(公告)号：EP3389672A1

公开(公告)日：2018-10-24

申请号：EP16876543.6

申请日：2016-12-14

申请人： COLD SPRING HARBOR LABORATORY ,  Stoke Therapeutics, Inc.

发明人： AZNAREZ, Isabel ,  NASH, Huw M. ,  HALL, Samuel W. ,  JING, Enxuan

IPC分类号： A61K31/7088 ,  C12N15/113 ,  C12Q1/68

CPC分类号： A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/321 ,  C12N2310/3521

摘要： Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a liver disease.

公开(公告)号：EP3353303A1

公开(公告)日：2018-08-01

申请号：EP16849742.8

申请日：2016-09-23

申请人： Ionis Pharmaceuticals, Inc. ,  Academisch Ziekenhuis Leiden h.o.d.n. LUMC

发明人： RIGO, Frank ,  PRAKASH, Thazha, P. ,  VAN ROON-MOM, Wilhelmina Maria, Clasina ,  TOONEN, Lodewijk Julius, Anton

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  A61K31/712 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/3525

摘要： Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.

公开(公告)号：EP3212658A4

公开(公告)日：2018-07-25

申请号：EP15855176

申请日：2015-10-30

申请人： COCRYSTAL PHARMA INC ,  UNIV EMORY

发明人： COATS STEVEN J ,  AMBLARD FRANCK ,  GARNIER-AMBLARD ETHEL ,  SCHINAZI RAYMOND F

IPC分类号： C07H19/10 ,  A61K31/712 ,  A61K38/21 ,  A61P31/12 ,  A61P35/00 ,  C07H19/067 ,  C07H19/167 ,  C07H19/20

CPC分类号： C07H19/10 ,  A61K31/7068 ,  A61K31/7072 ,  A61K31/712 ,  A61K38/21 ,  A61K45/06 ,  C07B59/005 ,  C07B2200/05 ,  C07H19/06 ,  C07H19/16 ,  C07H19/20 ,  Y02A50/385 ,  Y02A50/387 ,  Y02A50/393 ,  A61K2300/00

摘要： The present invention is directed to compounds, compositions and methods for treating or preventing Flaviviridae family of viruses (including HCV, Yellow fever, Dengue, Chikungunya Ebola and West Nile virus), RSV, HEV, and influenza infection and cancer in human subjects or other animal hosts.

公开(公告)号：EP2440183B1

公开(公告)日：2018-07-18

申请号：EP10786869.7

申请日：2010-06-10

申请人： Arbutus Biopharma Corporation

发明人： AKINC, Akin ,  DORKIN, Joseph, R. ,  QIN, Xiaojun ,  CANTLEY, William ,  MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil, G. ,  NARAYANANNAIR, Jayaprakash, K. ,  JAYARAMAN, Muthusamy ,  CHEN, Jianxin ,  ANSELL, Steven

IPC分类号： A61K9/127 ,  C07C229/12 ,  A61K31/221 ,  C12N15/113 ,  A61K48/00 ,  A61K47/14

CPC分类号： A61K9/1272 ,  A61K9/1278 ,  A61K31/22 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/713 ,  A61K38/1709 ,  A61K38/4846 ,  A61K47/14 ,  A61K47/549 ,  A61K47/551 ,  A61K47/6911 ,  A61K48/0033 ,  A61P9/00 ,  A61P25/28 ,  C07C29/58 ,  C07C227/18 ,  C07C227/40 ,  C07C229/12 ,  C07D475/04 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  Y02A50/463 ,  A61K2300/00 ,  C12N2310/3521 ,  C12N2310/3533

摘要： The invention features a cationic lipid of formula I, an improved lipid formulation comprising a cationic lipid of formula I and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.