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1. 发明申请

WO2016134375A1 METHODS AND COMPOSITIONS FOR TREATING GENETIC EYE DISEASES 审中-公开
标题翻译：用于治疗遗传性眼病的方法和组合物
公开(公告)号：WO2016134375A1
公开(公告)日：2016-08-25
申请号：PCT/US2016/018970
申请日：2016-02-22
申请人： UNIVERSITY OF IOWA RESEARCH FOUNDATION , FONDAZIONE TELETHON , DAVIDSON, Beverly, L. , CHEN, Yong Hong , AURICCHIO, Alberto
发明人： DAVIDSON, Beverly, L. , CHEN, Yong Hong , AURICCHIO, Alberto
IPC分类号： A01N43/04 , A01N63/00 , A01N65/00 , A61K48/00 , C12N15/63 , C12N15/85
CPC分类号： C12N15/8616 , A61K48/0008 , A61K48/0058 , A61P27/00 , C12N15/86 , C12N2750/14145 , C12N2810/50
摘要： The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents, to the eye. The present inventors have discovered peptides that function to target agents, such as viral vectors, to ocular cells. The present disclosure describes a method to utilize these novel peptides to direct, for example, viral capsids to the cell type of interest. In this instance, ocular cells (such as retinal cells) are targeted by the identified peptides. Vectors harboring capsid proteins modified to include such peptides can be used to provide therapeutic agents to the eye.
摘要翻译：本公开内容提供了靶向肽和载体，其含有将靶向递送药剂的肽的序列编码至眼睛。本发明人已经发现了将目标试剂（例如病毒载体）作用于眼细胞的肽。本公开内容描述了利用这些新型肽将例如病毒衣壳引导至感兴趣的细胞类型的方法。在这种情况下，眼细胞（如视网膜细胞）被鉴定的肽靶向。含有修饰成包括这些肽的衣壳蛋白的载体可用于向眼睛提供治疗剂。

2. 发明申请

WO2018225093A1 CHEMICAL COMPOUNDS AS ATF4 PATHWAY INHIBITORS 审中-公开
公开(公告)号：WO2018225093A1
公开(公告)日：2018-12-13
申请号：PCT/IN2018/050375
申请日：2018-06-07
申请人： GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LIMITED
发明人： AXTEN, Jeffrey Michael , DEMARTINO, Michael P. , EVANS, Karen Anderson , RALPH, Jeffrey M , KALITA, Biswajit
IPC分类号： C07D233/36 , C07C233/41 , C07D305/08 , C07D213/65 , A61K31/4166 , A61K31/337 , A61K31/44 , A61K31/165 , A61P35/00 , A61P25/00 , A61P9/00 , A61P3/00 , A61P27/00 , A61P29/00 , C07D311/58
CPC分类号： C07D213/65 , A61K31/4015 , A61K31/4412 , A61K31/4439 , A61K45/06 , A61P3/00 , A61P9/00 , A61P25/00 , A61P27/00 , A61P29/00 , A61P35/00 , C07C233/41 , C07C235/22 , C07C235/40 , C07C237/10 , C07C2601/02 , C07C2602/06 , C07C2602/10 , C07C2602/38 , C07C2602/40 , C07C2602/44 , C07D205/04 , C07D207/12 , C07D207/273 , C07D209/44 , C07D211/14 , C07D211/46 , C07D233/36 , C07D241/08 , C07D295/135 , C07D305/08 , C07D307/85 , C07D311/58 , C07D401/12 , A61K2300/00
摘要： The invention is directed to substituted bridged cycloalkane derivatives. Specifically, the invention is directed to compounds according to Formula (IIIQ): wherein X 6' , a, b, C 8' , D 8' , L 82' , L 83' , R 81' , R 82' , R 83' , R 84' , R 85' , R 86' , z 82' , z 84' , z 85' , and z 86' are as defined herein; or salts thereof. The compounds of the invention are inhibitors of the ATF4 pathway. Accordingly, invention is further directed to pharmaceutical compositions comprising a compound of the invention. The invention is still further directed to methods of inhibiting the ATF4 pathway and treatment of disorders associated therewith using a compound of the invention or a pharmaceutical composition comprising a compound of the invention.

3. 发明申请

WO2016094659A1 COMPOSITIONS AND METHODS FOR TREATING DISEASES AND CONDITIONS 审中-公开
标题翻译：用于治疗疾病和病症的组合物和方法
公开(公告)号：WO2016094659A1
公开(公告)日：2016-06-16
申请号：PCT/US2015/065000
申请日：2015-12-10
申请人： UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION , THE UNITED STATES GOVERNMENT AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS
发明人： CHEN, Beibei , MALLAMPALLI, Rama, K.
IPC分类号： C07C211/27 , C07C211/50 , C07C211/54 , C07D207/06 , C07D213/36 , C07D239/26 , C07D249/08 , C07D271/10 , C07D401/04 , C07D403/04 , A61K31/495 , A61K31/496 , A61K31/497 , A61K31/505 , A61P7/00 , A61P11/00 , A61P11/06 , A61P25/28 , A61P29/00 , A61P33/06 , A61P35/00
CPC分类号： C07D413/12 , A61K31/495 , A61K31/496 , A61K31/497 , A61K31/505 , A61P1/00 , A61P3/10 , A61P9/10 , A61P11/00 , A61P11/06 , A61P13/02 , A61P17/00 , A61P17/06 , A61P19/02 , A61P27/00 , A61P31/00 , A61P37/08 , C07C211/27 , C07C211/49 , C07C211/54 , C07C217/08 , C07C217/58 , C07C2601/14 , C07D239/26 , C07D241/12 , C07D413/14
摘要： A compound of Formula II, or a salt, ester, solvate, hydrate or prodrug thereof: where: X1 is a branched or unbranched C 1-10 alkyl, (CH 2 ) s -NH-(CH 2 ) t , (CH 2 ) s -0-(CH 2)t , or (CH 2 ) S -C(NH 2 )- (CH 2 ) v -NH-(CH 2 )t,where s, t and v are each, independently an integer from 1 to 5; A and B are each, independently, C 6-10 aryl, C 6-10 aryl-C 1-6 alkyl, C 3-9 heteroaryl, or C 3-9 heteroaryl-C 1- 6 alkyl, each optionally substituted with 1, 2 or 3 independently selected R g groups; C and D are each, independently, C 3-7 cycloalkyl, C 3-7 cycloalkyl-C 1-6 alkyl, C 3-7 heterocycloalkyl, C 3-7 heterocycloalkyl-C 1-6 alkyl, C 6-10 aryl, C 6-10 aryl-C 1-6 alkyl, C 3-9 heteroaryl, or C 3-9 heteroaryl-C 1-6 alkyl, each optionally substituted with 1, 2 or 3 independently selected R g groups; each R g is, independently, halogen, C 1-6 alkyl, C 1-6 haloalkyl, hydroxyl, C 1-6 alkoxy, C 1-6 haloalkoxy, amino, C 1-6 alkylamino, or di-C 1-6 alkylamino; and n 1 and p 1 are each, independently, integers from 1 to 10.
摘要翻译：式II的化合物或其盐，酯，溶剂合物，水合物或前药：其中：X 1是支链或非支链C 1-10烷基，（CH 2）s -NH-（CH 2）t，（CH 2）s-0 - （CH 2）t或（CH 2）SC（NH 2） - （CH 2）v -NH-（CH 2）t，其中s，t和v各自独立地为1至5的整数; A和B各自独立地为C 6-10芳基，C 6-10芳基-C 1-6烷基，C 3-9杂芳基或C 3-9杂芳基-C 1-6烷基，各自任选被1,2或3个独立选择的R g基团取代; C和D各自独立地为C 3-7环烷基，C 3-7环烷基-C 1-6烷基，C 3-7杂环烷基，C 3-7杂环烷基-C 1-6烷基，C 6-10芳基，C 6-10芳基-C 1-6烷基，C 3 -9个杂芳基或C 3-9杂芳基-C 1-6烷基，各自任选被1,2或3个独立选择的R g基团取代; 每个R g独立地为卤素，C 1-6烷基，C 1-6卤代烷基，羟基，C 1-6烷氧基，C 1-6卤代烷氧基，氨基，C 1-6烷基氨基或二-C 1-6烷基氨基; n1和p1各自独立地为1〜10的整数。

4. 发明申请

WO2010103291A2 TREATMENT OF DISEASES RELATED TO HYPERACTIVITY OF THE COMPLEMENT SYSTEM 审中-公开
标题翻译：治疗与补充系统的高血压有关的疾病
公开(公告)号：WO2010103291A2
公开(公告)日：2010-09-16
申请号：PCT/GB2010/000465
申请日：2010-03-12
申请人： CAMBRIDGE ENTERPRISE LIMITED , LACHMANN, Peter
发明人： LACHMANN, Peter
IPC分类号： A61K38/17
CPC分类号： A61K38/482 , A61K38/00 , A61K38/16 , A61K38/17 , A61K38/48 , A61P9/10 , A61P13/00 , A61P25/28 , A61P27/00 , C12N9/48
摘要： Raising the level of Factor I above physiological levels can be used to treat diseases in which the underlying pathology is linked to overactivity of the C3b-feedback cycle and the generation and pro-inflammatory effects of iC3b. Methods, agents, and compositions for treatment of such diseases are described.
摘要翻译：提高高于生理水平的因子I的水平可用于治疗潜在病理学与C3b反馈周期的过度活动以及iC3b的产生和促炎症作用相关的疾病。描述了用于治疗这些疾病的方法，试剂和组合物。

5. 发明申请

WO2018102715A1 COMPOSITIONS AND METHODS FOR TREATING AND/OR REDUCING CORNEAL DYSTROPHY 审中-公开
公开(公告)号：WO2018102715A1
公开(公告)日：2018-06-07
申请号：PCT/US2017/064264
申请日：2017-12-01
申请人： INDIANA UNIVERSITY RESEARCH AND TECHNOLOGY CORPORATION , PRICE, JR., Francis , FENG, Matthew
发明人： PRICE, JR., Francis , FENG, Matthew , WENLIN, Zhang , BONANNO, Joseph , LI, Hongde , TENNESSEN, Jason
IPC分类号： A61K9/00 , A61K35/00 , A61K38/00 , A61K38/18 , A61P17/02 , A61P27/00
CPC分类号： A61P17/02 , A61K31/194 , A61K31/198 , A61K31/7004 , A61P27/00 , A61K2300/00
摘要： Cornea endothelium is the posterior cell monolayer of cornea that maintains corneal clarity via its pump function. This paper discloses that glutamine metabolism plays a crucial role in maintaining the energy supply for corneal endothelium physiological pump function. This glutaminolysis is present in healthy cornea endothelium and conserved across species. And it is disrupted when there is loss of ammonia-linked SLC4A11 transporter function, in conditions like Congenital Hereditary Endothelial Dystrophy (CHED) and Fuch's Endothelial Corneal Dystrophy (FECD).

6. 发明申请

WO2016183585A1 RETINOL-BINDING PROTEIN 3 (RBP3) AS A PROTECTIVE FACTOR IN NON-DIABETIC RETINAL DEGENERATION 审中-公开
标题翻译：作为非糖尿病肾病变的保护因子的抗体结合蛋白3（RBP3）
公开(公告)号：WO2016183585A1
公开(公告)日：2016-11-17
申请号：PCT/US2016/032752
申请日：2016-05-16
申请人： JOSLIN DIABETES CENTER, INC.
发明人： KING, George Liang , KEENAN, Hillary A.
IPC分类号： A61K39/385 , A61P9/10 , A61P27/02 , A61P27/06
CPC分类号： A61K31/711 , A61K9/0048 , A61K31/7088 , A61K35/76 , A61K38/16 , A61K38/1709 , A61K45/06 , A61K48/005 , A61P27/00 , C12N15/86 , C12N15/861 , C12N2740/16043 , C12N2740/16071
摘要： Methods for increasing retinal thickness in a non-diabetic mammal, comprising administering to the mammal one or both of: (i) a composition comprising RBP3 polypeptide, and/or (ii) a composition comprising a nucleic acid encoding an RBP3 polypeptide.
摘要翻译：一种用于增加非糖尿病哺乳动物视网膜厚度的方法，包括向所述哺乳动物施用以下一种或两种：（i）包含RBP3多肽的组合物和/或（ii）包含编码RBP3多肽的核酸的组合物。

7. 发明申请

WO2011036202A1 NEUROPROTECTION RETINIENNE PAR DES INHIBITEURS DES CANAUX IONIQUES REGULES PAR LA SOUS-UNITE SUR 审中-公开
标题翻译：离子通道阻塞剂由地下管线调节后的神经保护作用
公开(公告)号：WO2011036202A1
公开(公告)日：2011-03-31
申请号：PCT/EP2010/064040
申请日：2010-09-23
申请人： ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS , POLAK, Michel , BERDUGO POLAK, Marianne , BEHAR-COHEN, Francine , CRISANTI LASSIAZ, Patricia
发明人： POLAK, Michel , BERDUGO POLAK, Marianne , BEHAR-COHEN, Francine , CRISANTI LASSIAZ, Patricia
IPC分类号： A61K31/198 , A61K31/403 , A61K31/445 , A61K31/64 , A61K45/06 , A61P27/00
CPC分类号： A61K31/64 , A61K31/195 , A61K31/198 , A61K31/403 , A61K31/445 , A61P27/00 , A61K2300/00
摘要： La présente invention concerne l'utilisation d'inhibiteurs des canaux ioniques régulés par la sous-unité SUR dans le traitement et/ou la prévention de pathologies oculaires associées à une ischémie et/ou une excitotoxicité rétinienne.
摘要翻译：本发明涉及由SUR亚基调节的离子通道阻断剂用于治疗和/或预防与缺血和/或视网膜兴奋性毒性相关的眼部疾病。

8. 发明申请

WO2019060994A1 COMPOUNDS FOR USE IN THE TREATMENT OR PREVENTION OF LOWE SYNDROME OR DENT DISEASE, AND METHODS THEREFOR 审中-公开
公开(公告)号：WO2019060994A1
公开(公告)日：2019-04-04
申请号：PCT/CA2018/051218
申请日：2018-09-27
申请人： UNIVERSITÉ DE MONTRÉAL , THE UNIVERSITY OF MANCHESTER , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , SORBONNE UNIVERSITÉ , INSTITUT PASTEUR
发明人： CARRENO, Sébastien , BEN EL KADHI, Khaled , ECHARD, Arnaud , CAUVIN, Clothilde , LOWE, Martin
IPC分类号： A61K31/18 , A61P13/12 , A61P3/00 , C07C311/21
CPC分类号： C07C311/21 , A61K31/18 , A61P3/00 , A61P13/12 , A61P27/00
摘要： Compounds, compositions and their use in the treatment or prevention of symptoms of Lowe Syndrome and Dent disease 2 are described, for instance, the use of such compounds in the treatment of Lowe Syndrome and Dent disease 2, or in the treatment or prevention of at least one symptom associated therewith. The compounds disclosed are of Formula I:

9. 发明申请

WO2019053584A1 DEVICE FOR INTRAOCULAR RELEASE OF A MEDICAMENT 审中-公开
公开(公告)号：WO2019053584A1
公开(公告)日：2019-03-21
申请号：PCT/IB2018/056907
申请日：2018-09-11
申请人： POLITECNICO DI MILANO
发明人： BOSCHETTI, Federica , FERRONI, Marco , CEREDA, Matteo Giuseppe
IPC分类号： A61F9/00 , A61K9/00
CPC分类号： A61F9/0017 , A61F2210/0004 , A61F2250/0068 , A61K9/0051 , A61K47/02 , A61P27/00
摘要： An implantable device (1) for scheduled intraocular drug delivery comprising at least two hollow solids inscribed one into the other, where the walls (2, 3, 4) of said at least two hollow solids are made of biodegradable material based on magnesium, and where the at least two hollow solids inscribed one into the other delimit a volume, called core (7), inside the innermost of the hollow solids and one or more volumes, called crowns (5, 6), comprised between the innermost hollow solid and the hollow solid at the exterior thereof, where the core and the at least one crown are adapted to house doses of at least one active ingredient, whereby the walls are impermeable continuous walls which become permeable as a function of the post- implantation time of said device.

10. 发明申请

WO2017160986A1 MESENCHYMAL STEM CELLS WITH ENHANCED EFFICACY 审中-公开
标题翻译：具有增强效力的间充质干细胞
公开(公告)号：WO2017160986A1
公开(公告)日：2017-09-21
申请号：PCT/US2017/022513
申请日：2017-03-15
申请人： CELL MEDICINE, INC.
发明人： RIORDAN, Neil
IPC分类号： A01N63/00 , A01N65/00
CPC分类号： A61K35/28 , A61P1/00 , A61P9/00 , A61P13/00 , A61P25/00 , A61P27/00 , A61P29/00 , C12N5/0663 , G01N33/4833 , G01N2333/70589 , G01N2333/70596
摘要： Disclosed are protocols, isolation means, and compositions of matter useful for identifying mesenchymal stem cells possessing enhanced clinical activity. In one embodiment, markers associated with said enhanced mesenchymal stem cell activity are utilized to identify donors whose mesenchymal stem cells possess superior efficacy compared to mesenchymal stem cells from donors who lack said markers associated with said enhanced efficacy. In one embodiment, said markers are utilized to select for mesenchymal stem cells possessing enhanced efficacy from in vitro cultures. In another embodiment, surfaces markers associated with said markers associated with enhanced efficacy are utilized to positively select for cells possessing enhanced efficacy. In another embodiment, the invention teaches markers whose expression is correlated with negative efficacy. Said markers can be utilized to exclude mesenchymal stem cell donors, or in vitro generated and/or isolated mesenchymal stem cells prior to clinical use.
摘要翻译：公开了用于鉴定具有增强临床活性的间充质干细胞的方案，分离手段和物质组成。在一个实施方案中，与所述增强的间充质干细胞活性相关的标记物被用于鉴定间充质干细胞与缺乏与所述增强的功效相关的所述标记物的供体的间充质干细胞相比具有优越功效的供体。在一个实施方案中，所述标记物用于从体外培养物中选择具有增强功效的间充质干细胞。在另一个实施方案中，利用与增强功效相关的所述标志物相关的表面标志物被用来积极选择具有增强功效的细胞。在另一个实施方案中，本发明教导了其表达与负面效力相关的标志物。所述标记物可用于在临床应用之前排除间充质干细胞供体或体外产生的和/或分离的间充质干细胞。

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