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71.发明公开SUGAR DERIVATIVES COMPRISING SULFUR-CONTAINING MOIETIES AND METHODS OF MAKING SAME AND METHODS OF USING THE SAME FOR THE TREATMENT OF MPS IIIC 审中-公开与硫含氧基团和方法糖衍生物用于其及其生产方法使用治疗MPS IIIC型
公开(公告)号：EP3105235A1
公开(公告)日：2016-12-21
申请号：EP15707462.6
申请日：2015-02-12
发明人： BOYD, Robert , LEE, Gary
IPC分类号： C07D497/04 , C07D498/04 , C07D513/04 , C07D211/46 , C07D211/56 , A61K31/429 , A61K31/435 , A61K31/4365 , A61K31/437 , A61P3/00
摘要： Described herein are modified sugar, iminosugar and azasugar compounds and methods of making same. One or more of these modified compounds contain sulfates, sulfites, sulfamates and/or sulfonamides. Also described are pharmaceutical compositions/formulations comprising these compounds, as well as methods using these modified sugar compounds for the treatment of MPS IIIC (also known as Sanfilippo Type C).
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72.发明授权UTILIZATION OF PHARMACOLOGICAL CHAPERONES TO IMPROVE MANUFACTURING AND PURIFICATION OF ACID BETA-GLUCOSIDASE 有权使用的药物分子伴侣提高了生产和清洁酸性β-葡萄糖苷
公开(公告)号：EP2435459B1
公开(公告)日：2016-05-04
申请号：EP10781143.2
申请日：2010-05-26
发明人： DO, Hung, V.
CPC分类号： C07D201/00 , C12N9/2402 , C12N9/96 , C12P21/02
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73.发明公开METHOD FOR TREATING ALZHEIMER'S DISEASE USING PHARMACOLOGICAL CHAPERONES TO INCREASE THE ACTIVITY OF GANGLIOSIDASES 有权阿尔茨海默氏病药理伴侣的治疗方法来增加GANGLIOSIDASEN的活度
公开(公告)号：EP2957295A1
公开(公告)日：2015-12-23
申请号：EP15173518.0
申请日：2010-10-12
CPC分类号： A61K31/7008 , A61K31/426 , A61K31/429 , A61K31/445 , A61K38/1709 , G06F19/00
摘要： The present invention relates to a method for treating an individual having Alzheimer's Disease by using pharmacological chaperones to increase the activity of gangliosidase and/or sialidase enzymes involved in ganglioside catabolism.
摘要翻译： 本发明涉及用于在个体中治疗通过使用药理学伴侣，以增加gangliosidase和/或分解代谢神经节苷脂参与唾液酸酶的酶的活性具有阿尔茨海默氏病的方法。
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74.发明公开METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES 审中-公开一种用于预测与药理伴侣疾病的治疗的反应
公开(公告)号：EP2946785A1
公开(公告)日：2015-11-25
申请号：EP15162630.6
申请日：2009-02-12
IPC分类号： A61K38/00 , G01N33/68 , C07K14/00 , A61K31/445 , C12Q1/34
CPC分类号： A61K31/445 , C07K14/00 , C12Q1/34 , G01N33/6893 , G01N2333/47 , G01N2333/94 , G01N2800/38 , G01N2800/52
摘要： A method of compiling a treatment reference table that indicates the responsiveness of one or more specific mutations of α-Gal A to a specific pharmacological chaperone, the method comprising: analysing in vitro the response to the specific pharmacological chaperone in a host cell that has been transformed with a nucleic acid vector that encodes a first mutant α-Gal A and compiling a table listing responsive and non-responsive mutant forms of α-Gal A. The specific pharmacological chaperone is 1-deoxygalactonojirimycin or a pharmaceutically salt or ester and the first cell is HEK-293 MSR cell.
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75.发明授权
公开(公告)号：EP2490533B1
公开(公告)日：2015-09-16
申请号：EP10825397.2
申请日：2010-10-05
发明人： BOYD, Robert , LEE, Gary , RYBCZYNSKI, Philip
IPC分类号： A01N43/40 , A61K31/445 , A61P3/00 , A61P43/00
CPC分类号： C07D211/46 , A61K31/15 , A61K31/445 , A61K38/47 , C07D211/96
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76.发明公开METHODS FOR COUPLING TARGETING PEPTIDES ONTO RECOMBINANT LYSOSOMAL ENZYMES FOR IMPROVED TREATMENTS OF LYSOSOMAL STORAGE DISEASES 有权METHOD FOR LINKING靶向的肽的重组溶酶体酶FOR改进的治疗溶酶体贮积病
公开(公告)号：EP2714752A4
公开(公告)日：2015-08-26
申请号：EP12793015
申请日：2012-05-25
发明人： DO HUNG
CPC分类号： A61K47/6847 , A61K38/47 , A61K47/48269 , A61K47/642 , C07K14/65 , C07K2319/00 , C07K2319/21 , C07K2319/50 , C12N9/16 , C12N9/2402 , C12N9/96
摘要： Described herein are methods of making targeting peptides conjugated to recombinant lysosomal enzymes by modifying the amino (N)-terminus and one or more lysine residues on recombinant human lysosomal enzymes using a first crosslinking agent to give rise to first crosslinking agent modified recombinant human lysosomal enzymes, modifying the first amino acid within a short linker at the amino (N)-terminus on a variant IGF-2 peptide using a second crosslinking agent to give rise to a second crosslinking agent modified variant IGF-2 peptide, and then conjugating the first crosslinking agent modified recombinant human lysosomal enzyme to the second crosslinking agent modified variant IGF-2 peptide containing a short linker. Also described herein are conjugates synthesized characterized as having higher affinities for the IGF2/CI-MPR receptor and cellular uptake using the methods disclosed herein. Also described herein are treatment methods using the disclosed conjugates.
摘要翻译： 描述中是通过修改氨基（N）末端和一个或上使用第一交联剂以产生第一交联剂修饰的重组人溶酶体酶的重组人溶酶体酶更多的赖氨酸残基使偶联到重组溶酶体酶靶向肽的方法 ，修改一个短左内的第一个氨基酸的氨基（N）末端上使用第二交联剂以产生第二交联剂的变体IGF-2肽修饰的变体IGF-2肽，然后缀合第一 交联剂修饰的重组人溶酶体酶与所述第二交联剂修饰的变体IGF-2肽含有短左侧。 因此，在描述的缀合物合成的特征在于具有用于IGF2 / CI-MPR受体和使用游离缺失中的方法的盘的细胞摄取更高的亲和力。 因此，在治疗方法中所描述使用的光盘共轭游离缺失。
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77.发明公开
公开(公告)号：EP2874648A1
公开(公告)日：2015-05-27
申请号：EP13820417.7
申请日：2013-07-16
CPC分类号： A61K38/47 , A61K31/445 , C12N9/2465 , C12Y302/01022 , A61K2300/00
摘要： The present application provides for compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an α-galactosidase A enzyme formulation.
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公开(公告)号：EP2533050B1
公开(公告)日：2014-03-12
申请号：EP12168451.8
申请日：2007-05-16
发明人： Lockhart, David , Castelli, Jeff
IPC分类号： G01N33/53 , G01N33/567 , G01N33/573 , A61K31/435
CPC分类号： A61K31/445 , A61K9/0053 , A61K9/48 , A61K31/45 , A61K45/06 , C07D211/46 , G01N33/5094 , G01N2333/924 , G01N2333/94 , G01N2800/04 , G01N2800/52 , A61K2300/00
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79.发明公开
公开(公告)号：EP2490533A4
公开(公告)日：2013-05-29
申请号：EP10825397
申请日：2010-10-05
发明人： BOYD ROBERT , LEE GARY , RYBCZYNSKI PHILIP
IPC分类号： A01N43/40 , A61K31/445 , A61P3/00 , A61P43/00
CPC分类号： C07D211/46 , A61K31/15 , A61K31/445 , A61K38/47 , C07D211/96
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公开(公告)号：EP1682504B1
公开(公告)日：2012-12-26
申请号：EP04810772.6
申请日：2004-11-12
发明人： FAN, Jian-Qiang , ZHU, Xiaoxiang , SHETH, Kamlesh
IPC分类号： C07D211/40 , A61K31/445
CPC分类号： C07D487/04
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