公开(公告)号：EP4389760A1

公开(公告)日：2024-06-26

申请号：EP22858844.8

申请日：2022-08-21

申请人： Joint Stock Company "Biocad"

发明人： STRELKOVA, Anna Nikolaevna ,  SHUGAEVA, Tatiana Evgenievna ,  GERSHOVICH, Pavel Mikhailovich ,  IAKOVLEV, Pavel Andreevich ,  MOROZOV, Dmitry Valentinovich

IPC分类号： C07K14/075 ,  C07K14/005 ,  C12N15/861 ,  C12N7/01 ,  C12N15/09 ,  A61K48/00

CPC分类号： A61K48/00 ,  C07K14/075 ,  C07K14/005 ,  C12N7/00 ,  C12N15/09 ,  C12N15/861

摘要： The present application relates to the fields of gene therapy and molecular biology. More specifically, the present invention relates to an isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9) comprising one or more amino acid substitutions compared to the wild-type AAV9 capsid protein VP1, which substitutions increase the efficiency of production (assembly) of the vector based on recombinant adeno-associated virus serotype 9 (rAAV9), to a capsid and a vector based on the above VP1, as well as to uses thereof.

公开(公告)号：EP2561067B1

公开(公告)日：2018-11-14

申请号：EP11726973.8

申请日：2011-04-22

申请人： University of Florida Research Foundation, Inc.

发明人： BOYE, Shannon Elizabeth ,  HAUSWIRTH, William W. ,  BOYE, Sanford Leon

IPC分类号： C12N7/00

CPC分类号： C12N15/861 ,  A61K38/00 ,  A61K48/005 ,  C12N9/88 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/85 ,  C12Y406/01002

摘要： Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).

公开(公告)号：EP3359662A1

公开(公告)日：2018-08-15

申请号：EP16854562.2

申请日：2016-10-11

申请人： The Children's Hospital of Philadelphia

发明人： DAVIDON, Beverly, L. ,  MONTEYS, Alejandro, Mas ,  EBANKS, Shauna

IPC分类号： C12N9/22 ,  C12N15/63 ,  C12N15/861 ,  C12N15/66

CPC分类号： C12N15/88 ,  C12N15/102 ,  C12N15/113 ,  C12N15/66 ,  C12N15/861 ,  C12N2310/20 ,  C12N2330/51

摘要： Compositions and methods are provided for the inhibition, treatment and/or prevention of Huntington's disease and related disorders.

公开(公告)号：EP3349760A1

公开(公告)日：2018-07-25

申请号：EP16847512

申请日：2016-09-17

申请人： CODA BIOTHERAPEUTICS INC

发明人： GREENBERG KENNETH P ,  DAVID NATHANIEL ,  FINER MITCHELL H

IPC分类号： A61K31/5513 ,  A61P3/04 ,  A61P25/14 ,  A61P25/28 ,  C12N15/861

CPC分类号： A61K38/177 ,  A61K9/0019 ,  A61K31/366 ,  A61K31/485 ,  A61K31/5513 ,  A61K38/08 ,  A61K38/10 ,  A61K38/1709 ,  A61K2300/00 ,  A61P25/16 ,  A61P25/28 ,  C07K14/705 ,  C07K14/72 ,  C12N15/861 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/002 ,  C12N2830/008

摘要： The present invention generally provides vectors, compositions, and methods of using the same for treating neurological disorders, including managing pain. The compositions and methods include the use of G protein-coupled receptors and ligand-gated ion channels to treat neurological indications including pain, epilepsy and satiety disorders. The compositions and methods further include the use of synthetic ligands to activate the G protein-coupled receptors and ligand-gated ion channels in the treatment of neurological disease.

公开(公告)号：EP3284826A4

公开(公告)日：2018-04-11

申请号：EP15889036

申请日：2015-11-27

申请人： GUANGZHOU N BIOMED CO LTD

发明人： CHEN LING ,  FENG LIQIANG

IPC分类号： C12N15/861

CPC分类号： C12N15/861 ,  C12N2710/10021 ,  C12N2710/10034 ,  C12N2710/10062

摘要： Provided are a human type 55 replication defective adenovirus vector, a method for preparing the same and uses thereof. The human type 55 replication defective adenovirus vector is prepared by the following method: knocking out E1 and E3 genes from Ad55, substituting the open reading frame 6 or the open reading frames 2, 3, 4, 6 and 6/7 of E4 gene in Ad55 genome with the corresponding open reading frames of Ad5 genome. In addition, an exogenous gene expression cassette may also be integrated into the E1 gene region of Ad55. Said human type 55 replication defective adenovirus vector is able to be produced in large scale in 293, PerC6 and other helper cell lines, and is able to be concentrated and purified by density gradient centrifugation. The human type 55 replication defective adenovirus vector is unable to replicate in normal human cells, thus has an attenuated phenotype, and the vector can express exogenous genes in target cells with high efficiency, thus can be used as vaccines or gene therapy vectors, as well as for drug and neutralizing antibody development and reporter-tracer system.

公开(公告)号：EP3230441A1

公开(公告)日：2017-10-18

申请号：EP15867378.0

申请日：2015-12-11

申请人： Voyager Therapeutics, Inc.

发明人： MCLAUGHLIN, James ,  KOTIN, Robert

IPC分类号： C12N7/02 ,  C12N15/861 ,  C12N15/864

CPC分类号： C12N15/861 ,  C07K14/005 ,  C12N7/00 ,  C12N7/02 ,  C12N15/86 ,  C12N15/864 ,  C12N2710/14144 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14151

摘要： The present invention is directed to viral vectors and methods of their production and use.

摘要翻译： 本发明涉及病毒载体及其生产和使用方法。

公开(公告)号：EP2649086B1

公开(公告)日：2017-07-19

申请号：EP11846757.0

申请日：2011-12-09

申请人： The Trustees of The University of Pennsylvania

发明人： JUNE, Carl, H. ,  LEVINE, Bruce, L. ,  PORTER, David, L. ,  KALOS, Michael, D. ,  MILONE, Michael C.

IPC分类号： A61K39/00 ,  A61K35/17

CPC分类号： C12N5/0636 ,  A61K35/17 ,  A61K38/177 ,  A61K38/1774 ,  A61K39/0011 ,  A61K39/39558 ,  A61K45/06 ,  A61K48/005 ,  A61K2039/505 ,  A61K2039/5156 ,  A61K2039/5158 ,  A61K2039/5256 ,  A61K2039/585 ,  C07K14/075 ,  C07K14/525 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70521 ,  C07K14/70578 ,  C07K14/70596 ,  C07K16/2803 ,  C07K16/2896 ,  C07K16/30 ,  C07K16/3061 ,  C07K2317/53 ,  C07K2317/622 ,  C07K2317/76 ,  C07K2317/80 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/30 ,  C07K2319/33 ,  C07K2319/74 ,  C12N7/00 ,  C12N15/85 ,  C12N15/861 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2510/00 ,  C12N2740/15034 ,  C12N2740/15043 ,  C12N2740/15071 ,  A61K2300/00

摘要： The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.

摘要翻译： 本发明提供了用于治疗人类癌症的组合物和方法。 本发明涉及施用遗传修饰的T细胞以表达CAR，其中CAR包含抗原结合结构域，跨膜结构域，共刺激信号传导区和CD3ζ信号传导结构域。

公开(公告)号：EP2649086A4

公开(公告)日：2014-12-31

申请号：EP11846757

申请日：2011-12-09

申请人： UNIV PENNSYLVANIA

发明人： JUNE CARL H ,  LEVINE BRUCE L ,  PORTER DAVID L ,  KALOS MICHAEL D ,  MILONE MICHAEL C

IPC分类号： C07H21/04 ,  A61K39/00

CPC分类号： C12N5/0636 ,  A61K35/17 ,  A61K38/177 ,  A61K38/1774 ,  A61K39/0011 ,  A61K39/39558 ,  A61K45/06 ,  A61K48/005 ,  A61K2039/505 ,  A61K2039/5156 ,  A61K2039/5158 ,  A61K2039/5256 ,  A61K2039/585 ,  C07K14/075 ,  C07K14/525 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70521 ,  C07K14/70578 ,  C07K14/70596 ,  C07K16/2803 ,  C07K16/2896 ,  C07K16/30 ,  C07K16/3061 ,  C07K2317/53 ,  C07K2317/622 ,  C07K2317/76 ,  C07K2317/80 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/30 ,  C07K2319/33 ,  C07K2319/74 ,  C12N7/00 ,  C12N15/85 ,  C12N15/861 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2510/00 ,  C12N2740/15034 ,  C12N2740/15043 ,  C12N2740/15071 ,  A61K2300/00

摘要： The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.

摘要翻译： 本发明提供了组合物和方法用于治疗人癌症。 本发明包括涉及施用遗传修饰的T细胞表达CAR worin抗原结合结构域的车厢包括，一个跨膜结构域，共刺激信号传导区域，和CD3ζ信号传导结构域。

公开(公告)号：EP3342865A1

公开(公告)日：2018-07-04

申请号：EP16887656.3

申请日：2016-10-24

申请人： Institute of Biotechnology, Academy of Military Medical Sciences, Pla.

发明人： CHEN, Wei ,  WU, Shipo ,  HOU, Lihua ,  SONG, Xiaohong ,  ZHANG, Jinlong ,  FU, Ling

IPC分类号： C12N15/40 ,  C12N15/861 ,  C12N7/01 ,  A61K39/12 ,  A61P31/14

CPC分类号： A61K39/12 ,  A61K2039/5254 ,  A61K2039/5256 ,  A61P31/14 ,  C07K14/005 ,  C12N15/86 ,  C12N15/861 ,  C12N2710/10041 ,  C12N2710/10062 ,  C12N2710/10343 ,  C12N2760/14122 ,  C12N2760/14134

摘要： Provided are an Ebola virus envelope glycoprotein (that is GP protein) codon optimized nucleotide sequence, a human replication deficient recombinant adenovirus capable of expressing the nucleotide sequence, and applications in preparing a vaccine for preventing Ebola virus diseases. The nucleotide sequence takes a replication deficient 5 type adenovirus that is lack of E1 and E3 in a combined mode as a vector, HEK293 cells that integrate adenovirus E1 genes serve as a packaging cell line, and carried protective antigenic genes are codon optimized Zaire type Ebola virus Makona strain envelope glycoprotein genes. After the envelope glycoprotein genes are optimized by codon, the expression level in transfection cells is obviously improved.

公开(公告)号：EP3305798A1

公开(公告)日：2018-04-11

申请号：EP17191702.4

申请日：2011-12-09

申请人： The Trustees of The University of Pennsylvania

发明人： JUNE, Carl H. ,  LEVINE, Bruce L. ,  PORTER, David L. ,  KALOS, Michael D. ,  MILONE, Michael C.

IPC分类号： C07H21/04 ,  A61K39/00

CPC分类号： C12N5/0636 ,  A61K35/17 ,  A61K38/177 ,  A61K38/1774 ,  A61K39/0011 ,  A61K39/39558 ,  A61K45/06 ,  A61K48/005 ,  A61K2039/505 ,  A61K2039/5156 ,  A61K2039/5158 ,  A61K2039/5256 ,  A61K2039/585 ,  C07K14/075 ,  C07K14/525 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70521 ,  C07K14/70578 ,  C07K14/70596 ,  C07K16/2803 ,  C07K16/2896 ,  C07K16/30 ,  C07K16/3061 ,  C07K2317/53 ,  C07K2317/622 ,  C07K2317/76 ,  C07K2317/80 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/30 ,  C07K2319/33 ,  C07K2319/74 ,  C12N7/00 ,  C12N15/85 ,  C12N15/861 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2510/00 ,  C12N2740/15034 ,  C12N2740/15043 ,  C12N2740/15071 ,  A61K2300/00

摘要： The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.