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1. 发明公开

EP3256116A4 BENZENESULFONAMIDE UPREGULATORS OF NPC1 FOR NEIMANN-PICK DISEASE AND OTHER LYSOSOMAL STORAGE DISORDERS 有权
公开(公告)号：EP3256116A4
公开(公告)日：2018-07-11
申请号：EP16749864
申请日：2016-02-11
申请人： ICAHN SCHOOL MED MOUNT SINAI , NAT INSTITUTES OF HEALTH
发明人： PATNAIK SAMARJIT , TAYLOR MERCEDES , CALVO RAUL ROLANDO , MARUGAN JUAN JOSE , SOUTHALL NOEL , ZHENG WEI , FERRER-ALEGRE MARC , DEHDASTHI SEAMEEN , DRANCHAK PATRICIA , CHEN FANNIE , IOANNOU YIANNIS
IPC分类号： A61K31/18 , A61K31/165 , A61P3/00 , A61P3/06
CPC分类号： C07C311/21 , A61K31/165 , A61K31/4164 , A61K31/44 , C07D207/335 , C07D213/46 , C07D233/64
摘要： Methods and compositions for treating lysosomal storage disorders are disclosed. The methods involve administering a genus of benzenesulfonamides, particularly N-[3-(aminosulfonyl)phenyl]-benzamides and heteroarylamides. A genus of suitable compounds is shown in formula I:

2. 发明公开

EP3149147A4 METHOD AND APPARATUS TO PREPARE CARDIAC ORGANOIDS IN A BIOREACTOR SYSTEM 有权
公开(公告)号：EP3149147A4
公开(公告)日：2018-05-02
申请号：EP15800086
申请日：2015-05-29
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： COSTA KEVIN DAVID , CASHMAN TIMOTHY JAMES , BACKERIS PETER CONSTANTINE
IPC分类号： C12M3/00 , C12N5/00
CPC分类号： C12M21/08 , C12M23/00 , C12M23/26 , C12M23/38 , C12M35/02
摘要： A bioreactor system for preparing a cardiac organoid chamber and for subsequent testing thereof is described herein and shown in the exemplary drawing figures. The bioreactor system includes a first vessel having a hollow interior and an open top. A first cover is mated with the open top of the first vessel. The first cover has a first opening formed therein. The system further includes a cannula having a lumen that extends from an open first end to an open second end. The cannula is disposed within the first opening of the first cover such that a portion of the cannula lies below the first cover and for insertion into the hollow interior of the first vessel. A porous ring is coupled to the cannula at or proximate the open second end thereof. The system also includes a balloon catheter having an inflatable balloon at a distal end of a catheter shaft. The balloon catheter is adapted to pass through the lumen of the cannula when the balloon is in a deflated state. The balloon catheter is axially adjustable within the lumen to allow the balloon in an inflated state to be disposed adjacent: (1) the open second end of the cannula; and (2) the porous ring for preparing the cardiac organoid chamber about the inflated balloon and porous ring. The cannula and porous ring construction and combination allows for the balloon to be deflated and removed from the lumen of the cannula while the engineered cardiac organoid chamber remains attached to the porous ring. This permits the testing of organoid pump function, such as organoid pressure and volume characteristics, without having to transfer the engineered cardiac organoid from one tool (e.g., an incubation tool) to another tool (e.g., a functional testing apparatus).

3. 发明公开

EP3148543A4 SMALL MOLECULE TRANSCRIPTION MODULATORS OF BROMODOMAINS 有权
公开(公告)号：EP3148543A4
公开(公告)日：2018-01-24
申请号：EP15800337
申请日：2015-05-29
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： ZHOU MING-MING , GERONA-NAVARRO GUILLERMO , RODRIGUEZ-FERNANDEZ YOEL , CASACCIA PATRIZIA
IPC分类号： A61K31/497 , A01N43/38 , A01N43/42 , A61K31/337 , A61K31/437 , A61K31/4745 , A61K31/475 , A61K31/704 , A61K31/7048 , A61K31/7068 , A61K45/06 , C07D217/00 , C07D471/04 , C07D487/00 , C12N5/071 , C12N5/079
CPC分类号： C07D471/04 , A61K31/337 , A61K31/437 , A61K31/4745 , A61K31/475 , A61K31/704 , A61K31/7048 , A61K31/7068 , A61K45/06 , C12N5/0602 , C12N5/0622 , C12N2501/999 , C12N2506/30 , A61K2300/00
摘要： This disclosure relates generally to compounds and compositions comprising one or more indole analogs. These compounds are useful for treating diseases associated with NF-kB and p53 activity, such as cancer and inflammatory diseases.

4. 发明公开

EP3043641A4 METHODS OF MAKING TRANSGENIC ANIMALS WITH T CELL RECEPTORS THAT ARE SPECIFIC TO A FLOURESCENT PROTEIN 有权
标题翻译：对于荧光蛋白特异性T细胞受体，转基因动物和它们的制备方法，分离的T细胞和方法使用
公开(公告)号：EP3043641A4
公开(公告)日：2017-06-07
申请号：EP14843371
申请日：2014-09-12
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： AGUDO JUDITH , BROWN BRIAN
IPC分类号： A01K67/00 , A01K67/033
CPC分类号： A01K67/0275 , A01K2217/30 , A01K2227/105 , A01K2267/0387 , A01K2267/0393 , C07K14/7051 , C12N15/8509 , G01N33/566 , G01N2333/70503
摘要： This invention relates to a transgenic non-human mammal whose genome comprises a polynucleotide sequence encoding a T cell receptor that is specific to a fluorescent protein, where the T cell of the non-human mammal comprises the T cell receptor. The present invention also relates to an isolated T cell from the transgenic non-human mammal of the present invention, an isolated T cell comprising an expression construct comprising a polynucleotide sequence that encodes a T cell receptor that is specific to a fluorescent protein, methods of making transgenic non-human mammals comprising T cell receptors that are specific to a fluorescent protein, a method of depleting cells in a non-human mammal using isolated T cells that encode a T cell receptor that is specific to a target protein, and a method of characterizing a T cell response to an agent.
摘要翻译：本发明涉及一种转基因非人类哺乳动物基因组中是谁的包含编码T细胞受体的多核苷酸序列确实是特定于荧光蛋白，其中非人类哺乳动物的T细胞包括T细胞受体。因此，本发明涉及从本发明的分离的T细胞的转基因非人类哺乳动物表达构建体包含含有的多核苷酸序列并编码T细胞受体的分离那样的T细胞是特定于荧光蛋白，的方法制备转基因非人类哺乳动物包含T细胞受体没有特定于荧光蛋白，使用分离的T细胞在非人类哺乳动物消耗细胞的方法没有编码T细胞受体确实是特异于靶蛋白，和一种方法的表征到的试剂的T细胞应答。

5. 发明公开

EP2983787A4 METHOD FOR TREATING POST-TRAUMATIC STRESS DISORDER 有权转让
标题翻译：一种用于治疗创伤后应激障碍
公开(公告)号：EP2983787A4
公开(公告)日：2017-01-18
申请号：EP14783146
申请日：2014-04-14
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： CHARNEY DENNIS S , FEDER ADRIANA
IPC分类号： A61P23/00 , A61K31/135
CPC分类号： A61K31/135 , A61K9/0019 , A61K9/0043 , A61K31/428 , A61K31/445 , A61K31/4525 , A61K31/517 , A61K31/53 , A61K33/00 , A61K45/06 , A61K2300/00
摘要： The present disclosure provides compositions containing ketamine and methods of using those compositions for the treatment of post-traumatic stress disorder. Also provided herein is a pharmaceutical composition that comprises esketamine and a pharmaceutically acceptable carrier, excipient or diluent, for use in treatment of PTSD. In some aspects, the pharmaceutical composition is for intranasal or intravenous administration. In some aspects, the pharmaceutical composition is for use in a method of treating PTSD in a subject. In some aspects, the pharmaceutical composition is for use in a method of treating major depressive disorder in a subject that is co-morbid with the PTSD.

6. 发明公开

EP3161158A4 METHODS FOR DIAGNOSING RISK OF RENAL ALLOGRAFT FIBROSIS AND REJECTION 有权转让
公开(公告)号：EP3161158A4
公开(公告)日：2018-03-07
申请号：EP15811195
申请日：2015-06-26
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： MURPHY BARBARA , ZHANG WEIJIA
IPC分类号： C12Q1/68 , G01N33/00
CPC分类号： C12Q1/6883 , A61K31/343 , A61K31/436 , A61K31/4412 , A61K31/52 , A61K31/5377 , A61K31/573 , A61K38/13 , A61K38/1774 , A61K38/18 , A61K38/1875 , A61K38/2221 , C07K14/70521 , C07K2319/30 , C12Q2600/118 , C12Q2600/158 , C12Q2600/178 , G01N2800/245 , G01N2800/347 , G01N2800/52
摘要： Disclosed herein is a method for diagnosing a renal allograft recipient's risk for developing fibrosis of the allograft and allograft loss. The method includes determining the expression levels of certain microRNAs, which have been determined to be predictive of an allograft recipient's risk. Also disclosed herein is a method of treating a renal allograft recipient to inhibit fibrosis of the allograft and allograft loss, as well as kits for use in the methods disclosed herein.

7. 发明公开

EP3074050A4 COMPOSITIONS AND METHODS FOR MODULATING NEURONAL EXCITABILITY AND MOTOR BEHAVIOR 有权
公开(公告)号：EP3074050A4
公开(公告)日：2017-11-29
申请号：EP14857152
申请日：2014-10-28
申请人： ICAHN SCHOOL MED MOUNT SINAI , UNIV ROCKEFELLER
发明人： SCHAEFER ANNE , GREENGARD PAUL
IPC分类号： C12N15/113 , A61K31/713 , A61P25/08
CPC分类号： C12N15/113 , A01K67/0275 , A01K67/0276 , A01K2217/052 , A01K2217/075 , A01K2217/203 , A01K2227/105 , A01K2267/0356 , A61K38/185 , A61K49/0008 , C12N2310/141 , C12N2310/315 , C12N2310/321 , C12N2310/341 , C12N2310/346 , C12N2310/3515 , C12N2310/3521 , G01N33/5058
摘要： The invention is directed to compositions and methods for treating or reducing the likelihood of the development of epilepsy in an individual. The method comprises administering to the central nervous system of an individual in need of such treatment a therapeutically effective amount of an agent capable of increasing the expression and/or activity of miR-128.

8. 发明公开

EP3126837A4 PEPTIDES, REAGENTS AND METHODS FOR DETECTING FOOD ALLERGY 无效转让
公开(公告)号：EP3126837A4
公开(公告)日：2017-11-22
申请号：EP15774062
申请日：2015-03-20
申请人： GENISPHERE LLC , ICAHN SCHOOL MED MOUNT SINAI
发明人： GETTS ROBERT C , KADUSHIN JAMES , SAMPSON HUGH A , BARDINA LUDA , GRISHINA GALINA , GIMENEZ GUSTAVO , LIN JING
IPC分类号： G01N33/68
CPC分类号： G01N33/564 , G01N33/6854 , G01N2800/24
摘要： Provided are peptide biomarkers for diagnosis of allergy, monitoring development of clinical tolerance in an allergic individual, and predicting whether an allergic subject is likely to develop clinical or natural tolerance over time. The invention also relates to diagnostic methods and diagnostic kits employing the peptide biomarkers.

9. 发明公开

EP3137071A4 USE OF 1,3-PROPANEDISULFONIC ACID OR PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF FOR THE TREATMENT OF SARCOIDOSIS 有权
公开(公告)号：EP3137071A4
公开(公告)日：2017-11-01
申请号：EP15786643
申请日：2015-04-29
申请人： ICAHN SCHOOL MED MOUNT SINAI
发明人： MORGENTHAU ADAM S
IPC分类号： A61K31/185 , A61K31/18 , A61K31/255 , A61K45/06 , A61P29/00 , A61P35/00
CPC分类号： A61K31/185 , A61K9/0053 , A61K45/06
摘要： The present disclosure relates to methods for the treatment of sarcoidosis. In certain aspects and embodiments, the disclosure provides compositions containing 1,3-propanedisulfonic acid or a pharmaceutically acceptable salt thereof and/or the use of such compositions for the treatment of Sarcoidosis. In another aspect, the disclosure relates to compositions containing 1,3-propanedisulfonic acid or a pharmaceutically acceptable salt thereof plus a second active agent. In yet another aspect, the disclosure relates to kits containing agents useful for the treatment of sarcoidosis.

10. 发明公开

EP2968525A4 NEWCASTLE DISEASE VIRUSES AND USES THEREOF 无效
标题翻译： MORBUS NEWCASTLE病毒及其用途
公开(公告)号：EP2968525A4
公开(公告)日：2016-10-26
申请号：EP14774247
申请日：2014-03-04
申请人： ICAHN SCHOOL MED MOUNT SINAI , SLOAN KETTERING INST CANCER
发明人： PALESE PETER , GARCIA-SASTRE ADOLFO , ZAMARIN DMITRIY , ALLISON JAMES , WOLCHOK JEDD D
IPC分类号： A61K39/17 , A61K35/768 , A61K39/00 , A61K39/395 , A61P35/00 , C07K16/28 , C12N7/00 , C12N15/09
CPC分类号： A61K35/768 , A61K39/3955 , A61K2039/505 , C07K16/2818 , C07K16/2827 , C07K16/2878 , C07K2317/76 , C12N7/00 , C12N2760/18121 , C12N2760/18122 , C12N2760/18132 , C12N2760/18133 , C12N2760/18143
摘要： Described herein are chimeric Newcastle disease viruses engineered to express an agonist of a co-stimulatory signal of an immune cell and compositions comprising such viruses. Also described herein are chimeric Newcastle disease viruses engineered to express an antagonist of an inhibitory signal of an immune cell and compositions comprising such viruses. The chimeric Newcastle disease viruses and compositions are useful in the treatment of cancer. In addition, described herein are methods for treating cancer comprising administering Newcastle disease viruses in combination with an agonist of a co-stimulatory signal of an immune and/or an antagonist of an inhibitory signal of an immune cell.

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