公开(公告)号：EP3710590B1

公开(公告)日：2024-07-17

申请号：EP18878835.0

申请日：2018-11-15

IPC分类号： C12N15/63 ,  A61K48/00 ,  C12N15/861 ,  A61P27/02

CPC分类号： A61K38/443 ,  A61P27/02 ,  C12Y101/01105 ,  C12N15/86 ,  C12N2750/1414320130101 ,  A01K2227/10520130101 ,  A01K2217/07520130101 ,  A01K2267/030620130101 ,  A61K48/005 ,  A61K48/0058

公开(公告)号：EP3773744B1

公开(公告)日：2024-05-22

申请号：EP19724606.9

申请日：2019-04-05

IPC分类号： A61K48/00 ,  C12N15/86 ,  C07K14/47 ,  A01K67/027

CPC分类号： C07K14/4728 ,  A01K67/0278 ,  A01K2217/07220130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  A61K48/005 ,  A61K48/0058 ,  C12N15/86 ,  C12N2750/1414320130101 ,  C12N2830/00820130101 ,  C12N2830/4820130101 ,  C12N2840/20320130101

公开(公告)号：EP4413990A2

公开(公告)日：2024-08-14

申请号：EP24168056.0

申请日：2018-05-17

申请人： The General Hospital Corporation

发明人： BREAKEFIELD, Xandra ,  MAGUIRE, Casey ,  PRABHAKAR, Shilpa ,  YELLEN, David

IPC分类号： A61K38/00

CPC分类号： A61K38/00 ,  A61K31/436 ,  A61K35/761 ,  A61K45/06 ,  A61K9/0019 ,  A61K48/005 ,  A01K2217/07520130101 ,  A01K2217/20620130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  C12N2740/1604320130101 ,  C12N2750/1414320130101 ,  C07K14/4705 ,  A61P35/00

摘要： The invention provides compositions and methods for treating tuberous sclerosis complex (TSC). In particular, provided are condensed tuberins (cTuberins), cTuberin nucleic acids, and recombinant adeno-associated viruses (rAAVs) carrying a cTuberin nucleic acid for treating a patient with TSC.

公开(公告)号：EP4364803A2

公开(公告)日：2024-05-08

申请号：EP24163209.0

申请日：2018-11-15

申请人： The Regents of the University of Michigan

发明人： THOMPSON, Debra A. ,  ALI, Robin R. ,  SMITH, Alexander J.

IPC分类号： A61P27/02

CPC分类号： A61K38/443 ,  A61P27/02 ,  C12Y101/01105 ,  C12N15/86 ,  C12N2750/1414320130101 ,  A01K2227/10520130101 ,  A01K2217/07520130101 ,  A01K2267/030620130101 ,  A61K48/005 ,  A61K48/0058

摘要： Provided are materials, methods and uses for treating an ophthalmological condition such as Leber Congenital Amaurosis by administering an effective amount of an adeno-associated virus AAV2, serotype 5 (AAV 2/5) or AAV-5 comprising an expressible coding region for human RDH12.

公开(公告)号：EP4361277A2

公开(公告)日：2024-05-01

申请号：EP24161028.6

申请日：2017-12-29

申请人： The Trustees of The University of Pennsylvania

发明人： WILSON, James, M. ,  SIDRANE, Jenny, Agnes ,  GOVINDASAMY, Lakshmanan

IPC分类号： C12N15/86

CPC分类号： C12N15/86 ,  C12N2750/1414320130101 ,  A01K2217/07520130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  A61P1/16 ,  A61P25/00 ,  A61P3/00

摘要： Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B.

公开(公告)号：EP4359006A1

公开(公告)日：2024-05-01

申请号：EP22744043.5

申请日：2022-06-22

申请人： Entrada Therapeutics, Inc.

发明人： SHEN, Xiulong ,  QIAN, Ziqing ,  DOUGHERTY, Patrick ,  KHEIRABADI, Mahboubeh ,  LI, Xiang

IPC分类号： A61K47/64 ,  C07K7/64 ,  C12N15/113

CPC分类号： A61K47/6455 ,  C07K7/64 ,  C12N15/113 ,  C12N2310/1120130101 ,  C12N2310/323320130101 ,  C12N2310/351320130101 ,  A61P21/00 ,  C07K19/00 ,  A01K2227/10520130101 ,  A01K2217/07220130101 ,  A01K2267/030620130101 ,  A61K38/00 ,  A61K31/712 ,  A61P25/00

公开(公告)号：EP4356727A2

公开(公告)日：2024-04-24

申请号：EP24160383.6

申请日：2019-04-05

申请人： Istituti Clinici Scientifici Maugeri S.p.A. S.B.

发明人： PRIORI, Silvia G. ,  MAZZANTI, Andrea ,  DENEGRI, Marco ,  NAPOLITANO, Carlo

IPC分类号： A01K67/027

CPC分类号： C07K14/4728 ,  A01K67/0278 ,  A01K2217/07220130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  A61K48/005 ,  A61K48/0058 ,  C12N15/86 ,  C12N2750/1414320130101 ,  C12N2830/00820130101 ,  C12N2830/4820130101 ,  C12N2840/20320130101

摘要： The disclosure features compositions and methods for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), particularly forms of the disease that are inherited in an autosomal dominant manner, by way of calsequestrin 2 (CASQ2) gene therapy. The compositions and methods described herein can be used to treat CPVT caused by mutations in, for example, ryanodine receptor 2 and calmodulin, such as calmodulin 1 (CALM1) and CALMS. The disclosure provides a variety of vectors that can be used for the delivery of a CASQ2 transgene to a patient, such as a human patient suffering from autosomal dominant CPVT, including adeno-associated virus vectors, among others.

公开(公告)号：EP4426107A1

公开(公告)日：2024-09-11

申请号：EP22823244.3

申请日：2022-11-04

申请人： Regeneron Pharmaceuticals, Inc.

发明人： STITT, Trevor ,  BRYDGES, Susannah ,  MUJICA, Alexander O. ,  ALLY, Roxanne

IPC分类号： A01K67/027 ,  C07K14/47

CPC分类号： A01K67/0275 ,  A01K2207/1520130101 ,  A01K2217/07220130101 ,  A01K2227/10520130101 ,  A01K2267/0220130101 ,  A01K2267/030620130101 ,  C07K14/47

公开(公告)号：EP4361277A3

公开(公告)日：2024-07-31

申请号：EP24161028.6

申请日：2017-12-29

申请人： The Trustees of The University of Pennsylvania

发明人： WILSON, James, M. ,  SIDRANE, Jenny, Agnes ,  GOVINDASAMY, Lakshmanan

IPC分类号： A61K48/00 ,  C07K14/755 ,  C12N15/86

CPC分类号： C12N15/86 ,  C12N2750/1414320130101 ,  A01K2217/07520130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  A61P1/16 ,  A61P25/00 ,  A61P3/00

摘要： Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B.

公开(公告)号：EP3390644B1

公开(公告)日：2024-07-31

申请号：EP16822578.7

申请日：2016-12-14

IPC分类号： C12N15/86 ,  A01K67/0276 ,  C12N9/12 ,  A61K48/00 ,  A61P43/00 ,  A61P31/00 ,  A61P25/28 ,  A61P21/00 ,  A61P11/16 ,  A61P11/00 ,  A61P19/00 ,  A61P1/10 ,  A61P1/12 ,  A61P9/00

CPC分类号： A01K67/0276 ,  A61K48/005 ,  C12N9/1288 ,  C12N15/86 ,  A01K2217/07520130101 ,  A01K2227/10520130101 ,  A01K2267/030620130101 ,  C12N2750/1414320130101 ,  C12N2830/1520130101 ,  C12N2830/5020130101 ,  A61P1/10 ,  A61P1/12 ,  A61P11/00 ,  A61P11/16 ,  A61P19/00 ,  A61P21/00 ,  A61P25/28 ,  A61P31/00 ,  A61P43/00 ,  A61P9/00