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41. 发明申请

US20170015987A1 MODIFIED CASPASE POLYPEPTIDES AND USES THEREOF 有权
标题翻译：改性CASPASE POLYPEPTIDES及其用途
公开(公告)号：US20170015987A1
公开(公告)日：2017-01-19
申请号：US15224441
申请日：2016-07-29
申请人： Bellicum Pharmaceuticals, Inc.
发明人： David SPENCER , Wei-Chun Chang
IPC分类号： C12N9/64 , A61K38/52 , C12N9/90 , A61K38/48
CPC分类号： C12N9/6472 , A61K31/4545 , A61K38/4873 , A61K38/52 , C12N9/90 , C12Y304/22062 , C12Y502/01008
摘要： The technology relates in part to compositions comprising modified Caspase-9 polypeptides, compositions comprising nucleic acids coding for modified Caspase-9 polypeptides, chimeric modified Caspase-9 polypeptides, and methods of use thereof, including methods for cell therapy. Methods for cell therapy include modifying transfused cells to express an inducible modified Caspase-9 protein, with reduced basal activity in the absence of the inducer.
摘要翻译：该技术部分地涉及包含修饰的半胱天冬酶-9多肽的组合物，包含编码修饰的半胱天冬酶-9多肽的核酸，嵌合修饰的半胱天冬酶-9多肽的组合物及其使用方法，包括细胞治疗方法。细胞治疗方法包括修改输血细胞以表达诱导型修饰的半胱天冬酶-9蛋白，在不存在诱导物的情况下具有降低的基础活性。

42. 发明授权

US09433688B2 Method of treating or retarding the development of blindness 有权
标题翻译：治疗或延缓失明发展的方法
公开(公告)号：US09433688B2
公开(公告)日：2016-09-06
申请号：US14310015
申请日：2014-06-20
申请人： The Trustees of the University of Pennsylvania , University of Florida Research Foundation, Incorporated , Cornell Research Foundation, Inc.
发明人： Gregory M. Acland , Gustavo D. Aguirre , Jean Bennett , William W. Hauswirth , Samuel G. Jacobson , Albert M. Maguire
IPC分类号： A61K48/00 , C12N15/12 , C12N15/861 , A61K31/70 , C07K14/705 , C12N15/86 , A61K38/51 , A61K38/52 , C12N15/864 , C12N7/00 , A61K38/00
CPC分类号： A61K48/0058 , A61K9/0048 , A61K31/70 , A61K38/00 , A61K38/465 , A61K38/51 , A61K38/52 , A61K48/00 , A61K48/005 , A61K48/0075 , C07K14/705 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14132 , C12N2750/14142 , C12N2750/14143 , C12N2750/14171 , C12N2830/008 , C12N2830/85 , C12N2840/203 , C12Y301/01064
摘要： A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.
摘要翻译：用于治疗人或动物对象的眼细胞中缺陷或不存在正常基因的眼部疾病的方法包括通过视网膜下注射给予受试者有效量的携带核酸序列的重组腺相关病毒编码在表达基因在眼细胞中的产物的启动子序列控制下的正常基因。眼细胞优选为视网膜色素上皮细胞（RPE），该基因优选为RPE特异性基因，例如RPE65。启动子是可以在RPE细胞中表达基因产物的启动子。视网膜下给药的组合物在该方法中是有用的。

43. 发明授权

US09399070B2 Compositions comprising a polymeric reagent and a pharmacologically active agent 有权
标题翻译：包含聚合物试剂和药理活性剂的组合物
公开(公告)号：US09399070B2
公开(公告)日：2016-07-26
申请号：US14720213
申请日：2015-05-22
申请人： Nektar Therapeutics
发明人： J. Milton Harris , Antoni Kozlowski , Samuel P. McManus , Michael D. Bentley , Stephen A. Charles
IPC分类号： A61K47/48 , A61K38/11 , A61K38/52 , A61K38/18 , A61K38/19 , A61K38/21 , A61K38/24 , A61K38/27 , A61K38/37 , A61K31/357 , C08G65/333 , C08G65/329 , A61K31/52 , A61K31/4025
CPC分类号： A61K47/60 , A61K31/357 , A61K31/4025 , A61K31/52 , A61K38/11 , A61K38/1816 , A61K38/193 , A61K38/212 , A61K38/215 , A61K38/24 , A61K38/27 , A61K38/37 , C08G65/329 , C08G65/33337 , C08G65/33344
摘要： Pharmaceutical compositions of conjugates are provided. The conjugates can be prepared by (among other ways) conjugating a pharmacologically active agent with a polymeric reagent that has a particular arrangement of atoms. Related methods, preparations, and so forth are also provided.
摘要翻译：提供了缀合物的药物组合物。缀合物可以通过使药理活性剂与具有特定原子排列的聚合物试剂（其他方式）来制备。还提供了相关的方法，准备等。

44. 发明申请

US20160114005A1 HYBRID PROTEIN THAT CONVERTS ARACHIDONIC ACID INTO PROSTACYCLIN 审中-公开
标题翻译：将ARACHIDONIC酸转化为PROSTACYCLIN的混合蛋白质
公开(公告)号：US20160114005A1
公开(公告)日：2016-04-28
申请号：US14885561
申请日：2015-10-16
申请人： UNIVERSITY OF HOUSTON SYSTEM
发明人： Ke-He RUAN
IPC分类号： A61K38/44 , C12N9/02 , C12N9/90 , C07K14/72 , A61K38/52
CPC分类号： A61K38/44 , A61K38/52 , C07K14/723 , C07K2319/00 , C07K2319/03 , C07K2319/70 , C12N9/0083 , C12N9/90 , C12Y114/99001 , C12Y503/99004
摘要： A recombinant 130-kDa protein is constructed by linking together human cyclooxygenase (COX) isoform-2 (COX-2) and prostacyclin synthase (PGIS), via a 10-20 amino acid residues of a transmembrane sequence. The engineered protein is expressed in cells, and adopts the functions of COX and PGIS, to continually convert arachidonic acid (AA) into prostaglandin G2 (catalytic step 1), prostaglandin H2 (catalytic step 2) and prostacyclin (PGI2; catalytic step 3).
摘要翻译：通过跨膜序列的10-20个氨基酸残基将人环加氧酶（COX）异构体-2（COX-2）和前列环素合酶（PGIS））连接在一起构建重组的130-kDa蛋白质。工程蛋白在细胞中表达，采用COX和PGIS的功能，将花生四烯酸（AA）连续转化为前列腺素G2（催化步骤1），前列腺素H2（催化步骤2）和前列环素（PGI2;催化步骤3）。

45. 发明授权

US09289474B2 Agent for use in the case of fructose intolerance 有权
公开(公告)号：US09289474B2
公开(公告)日：2016-03-22
申请号：US13833092
申请日：2013-03-15
申请人： Daniel Henry Wyrobnik , Isaac Harry Wyrobnik
发明人： Daniel Henry Wyrobnik , Isaac Harry Wyrobnik
IPC分类号： C12N9/92 , A61K38/52 , A61K38/54 , A23L1/305 , A23L1/03 , A23L1/30 , A61K38/44 , A61K9/48
CPC分类号： A61K38/52 , A23L29/06 , A23L33/10 , A23L33/18 , A23L33/20 , A23V2002/00 , A61K9/485 , A61K9/4858 , A61K38/44 , A61K38/54 , A61K2300/00 , A61P1/00 , C12Y101/99011 , C12Y302/0102 , C12Y302/01026 , C12Y503/01005
摘要： There is provided in accordance with embodiments of the invention a method of treating or reducing the effects in a subject of a condition selected from fructose intolerance and impaired fructose metabolism, the method comprising administering to a subject in need of such treatment or reduction an efficacious amount of a glucose isomerase, other than in combination with 5-D-fructose dehydrogenase. Other embodiments are also disclosed.

46. 发明申请

US20140030244A1 EXTRACTS ISOLATED FROM ELECTROPORATED AMBHIBIAN OOCYTES AND USE THEREOF IN TREATING DISEASES AND DISORDERS 审中-公开
标题翻译：从电解阿布肝类药物中分离的提取物及其在治疗疾病和病症中的应用
公开(公告)号：US20140030244A1
公开(公告)日：2014-01-30
申请号：US13949977
申请日：2013-07-24
申请人： BIOQUARK, INC.
发明人： Sergei Paylian
IPC分类号： A61K35/56 , A61K31/7105 , A61K38/52 , A61K38/45 , A61K38/46
CPC分类号： A61K35/65 , A61K31/7105 , A61K38/1703 , A61K38/45 , A61K38/46 , A61K38/52 , C12Y207/04006 , C12Y303/01001 , C12Y503/99002 , C12Y504/02002 , A61K2300/00
摘要： The described invention provides methods for preparing a composition containing extracts of activated amphibian oocytes, the method where the composition is a pharmaceutical composition comprising an equal volume of the extra-oocyte composition and the intra-oocyte composition, and a method for treating a disease, disorder, condition or injury characterized by a damaged or a cancerous differentiated cell including: (a) preparing the composition by the described method; (b) formulating a pharmaceutical composition comprising an equal volume of the extra-oocyte composition and the intra-oocyte composition, and optionally a carrier; and (c) administering a therapeutic amount of the pharmaceutical composition of (b) to a subject in need thereof, where the therapeutic amount is effective to reprogram the damaged or cancerous cells into iPSC-like cells capable of differentiating into cells capable of repairing the damaged or cancerous cells, thereby treating the disease, disorder, injury or condition.
摘要翻译：所述发明提供了制备含有活化的两栖动物卵母细胞提取物的组合物的方法，所述组合物是包含等体积卵母细胞组合物和卵母细胞组合物的药物组合物的方法，以及治疗疾病的方法，特征在于损伤或癌分化细胞的疾病，病症或损伤，包括：（a）通过所述方法制备组合物; （b）配制包含等体积卵母细胞组合物和卵母细胞组合物以及任选的载体的药物组合物; 和（c）将治疗量的（b）的药物组合物施用于有需要的受试者，其中治疗量有效地将损伤或癌细胞重编程为能够分化成能够修复细胞的细胞的iPSC样细胞损伤或癌细胞，从而治疗疾病，障碍，损伤或病症。

47. 发明申请

US20130209488A1 METHODS AND COMPOSITIONS FOR MALIC ENZYME 2 (ME2) AS A TARGET FOR CANCER THERAPY 有权
标题翻译：作为癌症治疗目标的MALIC ENZYME 2（ME2）的方法和组合
公开(公告)号：US20130209488A1
公开(公告)日：2013-08-15
申请号：US13814614
申请日：2011-08-05
申请人： Vikas P. Sukhatme , Jian-Guo Ren
发明人： Vikas P. Sukhatme , Jian-Guo Ren
IPC分类号： A61K39/395 , A61K31/56 , A61K45/06 , A61K38/52 , A61K31/336 , A61K33/24 , A61K31/282 , A61K31/196 , A61K31/506 , A61K31/7105 , A61K31/7052
CPC分类号： A61K39/3955 , A61K31/196 , A61K31/198 , A61K31/282 , A61K31/335 , A61K31/336 , A61K31/337 , A61K31/506 , A61K31/555 , A61K31/56 , A61K31/7052 , A61K31/7105 , A61K31/713 , A61K33/00 , A61K33/24 , A61K38/52 , A61K45/06 , C12N15/1137 , C12N2330/51 , C12Y101/01037 , G01N33/57484 , G01N2333/904 , G01N2500/10 , C12N2310/14 , C12N2310/531
摘要： The present invention relates to methods, compositions, and diagnostic tests for treating and diagnosing cancer and other related diseases that result in dysregulation of malic enzyme 2. In particular, the methods and compositions include combination therapy, such as with a combination of two or more ME2 inhibitors or a combination of an ME2 inhibitor and an anticancer agent.
摘要翻译：本发明涉及用于治疗和诊断导致苹果酶2失调的癌症和其它相关疾病的方法，组合物和诊断测试。特别地，所述方法和组合物包括组合疗法，例如两种或多种 ME2抑制剂或ME2抑制剂与抗癌剂的组合。

48. 发明申请

US20120156221A1 Method and Pharmaceutical Composition for Use in the Treatment of Neurodegenerative Disorders 审中-公开
标题翻译：用于治疗神经退行性疾病的方法和药物组合物
公开(公告)号：US20120156221A1
公开(公告)日：2012-06-21
申请号：US13379007
申请日：2010-06-18
申请人： Ludivine Perrocheau , Alain Van Dorsselaer , Jose Alain Sahel , Thierry Leveillard
发明人： Ludivine Perrocheau , Alain Van Dorsselaer , Jose Alain Sahel , Thierry Leveillard
IPC分类号： A61K39/395 , A61K38/17 , A61P27/02 , G01N33/566 , A61P25/28 , A61K31/7088 , A61K38/52
CPC分类号： G01N33/5058 , A61K38/52 , G01N33/5023 , G01N2333/70596 , G01N2800/28
摘要： The invention relates to compounds which activate the BASIGIN signaling pathway, preferably agonists of BASIGIN, for the treatment of neurodegenerative disorders.
摘要翻译：本发明涉及活化BASIGIN信号通路的化合物，优选BASIGIN的激动剂用于治疗神经变性疾病。

49. 发明申请

US20100284990A1 Compositions and Methods for Making Therapies Delivered by Viral Vectors Reversible for Safety and Allele-Specificity 有权
标题翻译：由病毒载体产生治疗的组合物和方法可逆的安全性和等位基因特异性
公开(公告)号：US20100284990A1
公开(公告)日：2010-11-11
申请号：US12580996
申请日：2009-10-16
申请人： William F. Kaemmerer , Eric Burright , Deepak Ramesh Thakker , Jennifer Heisel
发明人： William F. Kaemmerer , Eric Burright , Deepak Ramesh Thakker , Jennifer Heisel
IPC分类号： A61K38/52 , C07H21/04 , C12N15/63 , A61P25/00
CPC分类号： A61K31/70 , A61K48/005 , A61K48/0066 , C12N2799/025 , C12N2800/30
摘要： The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies.
摘要翻译：本发明涉及用于调节亨廷顿病基因疗法的组合物，方法和试剂盒，包括但不限于可逆基因治疗和等位基因特异性疗法。

50. 发明申请

US20090311232A1 Agent for use in the case of fructose intolerance 有权
标题翻译：在果糖不耐受的情况下使用的代理
公开(公告)号：US20090311232A1
公开(公告)日：2009-12-17
申请号：US12544242
申请日：2009-08-20
申请人： Daniel Henry Wyrobnik , Isaac Harry Wyrobnik
发明人： Daniel Henry Wyrobnik , Isaac Harry Wyrobnik
IPC分类号： A61K38/52 , C12N9/92
CPC分类号： A61K38/52 , A23L29/06 , A23L33/10 , A23L33/18 , A23L33/20 , A23V2002/00 , A61K9/485 , A61K9/4858 , A61K38/44 , A61K38/54 , A61K2300/00 , A61P1/00 , C12Y101/99011 , C12Y302/0102 , C12Y302/01026 , C12Y503/01005
摘要： There is provided in accordance with embodiments of the invention a method of treating or reducing the effects in a subject of a condition selected from fructose intolerance and impaired fructose metabolism, the method comprising administering to a subject in need of such treatment or reduction an efficacious amount of a glucose isomerase, other than in combination with 5-D-fructose dehydrogenase. Other embodiments are also disclosed.
摘要翻译：根据本发明的实施方案提供了一种治疗或降低受试者中从果糖不耐受和果糖代谢受损的病症的作用的方法，所述方法包括向需要这种治疗或减少的受试者施用有效量的葡萄糖异构酶，除了与5-D-果糖脱氢酶组合。还公开了其他实施例。

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