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1. 发明申请

WO2019005993A1 METHODS FOR TREATING HUNTINGTON'S DISEASE 审中-公开
公开(公告)号：WO2019005993A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/039794
申请日：2018-06-27
申请人： PTC THERAPEUTICS, INC.
发明人： BHATTACHARYYA, Anuradha , JANI, Minakshi, B.
IPC分类号： A61P25/28 , A61K31/517 , C07D239/70 , C07D403/14
摘要： The present description relates to a method or use of a compound for treating or ameliorating HD (Huntington's Disease) in a subject in need thereof comprising administering to the subject an effective amount of a compound of Formula (I): or a form thereof, wherein R 1 , R 2 , R a , R b and R c are as defined herein. In particular, the present description relates to a method of use of a compound of Formula (I) or a form or composition thereof for treating or ameliorating HD in a subject in need thereof comprising, administering an effective amount of the compound or a form or composition thereof to the subject.

2. 发明申请

WO2018226622A1 COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE 审中-公开
公开(公告)号：WO2018226622A1
公开(公告)日：2018-12-13
申请号：PCT/US2018/035954
申请日：2018-06-05
申请人： PTC THERAPEUTICS, INC.
发明人： WOLL, Matthew, G. , AMEDZO, Lukiana , BABU, Suresh , BARRAZA, Scott, J. , BHATTACHARYYA, Anuradha , KARP, Gary, Mitchell , MAZZOTTI, Anthony, R. , NARASIMHAN, Jana , PATEL, Jigar , TURPOFF, Anthony , XU, Zhenrong
IPC分类号： C07D239/74 , C07D239/94 , C07D239/95
摘要： The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

3. 发明申请

WO2015076800A1 SUBSTITUTED PYRIDINE AND PYRAZINE BMI-1 INHIBITORS 审中-公开
标题翻译：取代的吡啶和吡嗪BMI-1抑制剂
公开(公告)号：WO2015076800A1
公开(公告)日：2015-05-28
申请号：PCT/US2013/071142
申请日：2013-11-21
申请人： PTC THERAPEUTICS, INC.
发明人： BAIAZITOV, Ramil , CHOI, Soongyu , DU, Wu , HWANG, Seongwoo , LEE, Chang-Sun , LIU, Ronggang , MOON, Young-Choon , PAGET, Steven, D. , REN, Hongyu , SYDORENKO, Nadiya , WILDE, Richard, Gerald
IPC分类号： C07D241/20
CPC分类号： C07D403/04 , A61K31/497 , A61K31/4985 , A61K31/519 , A61K31/69 , A61K45/06 , C07D401/04 , C07D401/14 , C07D403/14 , C07D405/14 , C07D413/04 , C07D417/14 , C07D471/04 , C07D487/04 , C07D513/04 , C07F5/04
摘要： Amine substituted pyridine and pyrazine compounds and forms thereof that inhibit the function and reduce the level of B -cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.
摘要翻译：胺取代的吡啶和吡嗪化合物及其形式，其抑制B细胞特异性莫洛尼鼠白血病病毒整合位点1（Bmi-1）蛋白的功能和水平降低，以及它们用于抑制Bmi-1功能和降低水平的方法的Bmi-1治疗由Bmi-1介导的癌症。

4. 发明申请

WO2010022050A1 METHOD FOR IMPROVING UNIFORMITY OF CONTENT IN EDIBLE FILM MANUFACTURING 审中-公开
标题翻译：用于改善可食电影制造中内容的均匀性的方法
公开(公告)号：WO2010022050A1
公开(公告)日：2010-02-25
申请号：PCT/US2009/054162
申请日：2009-08-18
申请人： MONOSOL RX, LLC , MYERS, Garry , HARIHARAN, Madhusudan , SANGHVI, Pradeep , SZOPINSKI, Alex , DAILEY, Patrick
发明人： MYERS, Garry , HARIHARAN, Madhusudan , SANGHVI, Pradeep , SZOPINSKI, Alex , DAILEY, Patrick
IPC分类号： B32B1/00
CPC分类号： A61K9/7007 , A23P30/00 , A61K31/485
摘要： The present invention relates to a method of making a film composition with an improved uniformity of active composition The invention includes a method including the steps of forming a flowable film composition comprising an edible water-soluble polymer, a polar solvent and a particulate active suspended therein, where the ratio of solids to liquids is at least about 95 to about 5 and where the composition compπses a mixture having compositional uniformity per unit volume, directing the flowable film composition to a transfer trough, the transfer trough including a first rotating roller and a movable transfer substrate upon which to form the film, and maintaining uniformity of the flowable film composition dunng the residence time in the trough by maintaining the rate of composition enteπng the transfer trough and the level of the flowable film composition in the transfer trough at a substantially constant level
摘要翻译：本发明涉及一种制备具有改善的活性组合物均匀性的薄膜组合物的方法。本发明包括一种方法，其包括以下步骤：形成包含食用水溶性聚合物，极性溶剂和悬浮在其中的颗粒活性物质的可流动薄膜组合物其中固体与液体的比率为至少约95至约5，并且其中组合物压制具有每单位体积的组成均匀性的混合物，将可流动膜组合物引导至转移槽，转移槽包括第一旋转辊和可移动的转移基底，在其上形成膜，并且通过保持转移槽的组成速率和转移槽中的可流动的膜组合物的水平基本上保持在可移动的膜组合物的均匀性，阻止在槽中的停留时间恒定水平

5. 发明申请

WO2010002987A1 METHODS FOR SCREENING FOR COMPOUNDS FOR TREATING CANCER 审中-公开
标题翻译：筛选化合物治疗癌症的方法
公开(公告)号：WO2010002987A1
公开(公告)日：2010-01-07
申请号：PCT/US2009/049399
申请日：2009-07-01
申请人： PTC THERAPEUTICS, INC , CAO, Liangxian , DAVIS, Thomas
发明人： CAO, Liangxian , DAVIS, Thomas
IPC分类号： C12Q1/68 , C12N15/00 , C40B30/02
CPC分类号： C12Q1/6897
摘要： The methods described herein provide nucleic acid constructs and screening methods for identifying and validating compounds for use in the treatment of cancer, wherein the compounds down-regulate the post-transcriptional expression of Bmi-1.
摘要翻译：本文描述的方法提供用于鉴定和验证用于治疗癌症的化合物的核酸构建体和筛选方法，其中所述化合物下调Bmi-1的转录后表达。

6. 发明申请

WO2008147772A1 FILM SHREDS AND DELIVERY SYSTEMS INCORPORATING SAME 审中-公开
标题翻译：电影胶卷和交付系统
公开(公告)号：WO2008147772A1
公开(公告)日：2008-12-04
申请号：PCT/US2008/064245
申请日：2008-05-20
申请人： MONOSOL RX, LLC , SANGHVI, Pradeep , FUISZ, Richard, C.
发明人： SANGHVI, Pradeep , FUISZ, Richard, C.
IPC分类号： A61K9/00
CPC分类号： A61K31/465 , A61K9/006 , A61K9/1617 , A61K9/1623 , A61K9/1635 , A61K9/1652 , A61K9/1664 , A61K9/7007
摘要： Compositions are provided that include water soluble or water dispersible film shreds, a carrier and an active, such as, but not limited to a drug, flavoring agent or coloring agent. Also provided are methods of use of the film shreds, such as in improving the dissolution, stability, aesthetics, or taste of a composition or its components, and in reducing a disease risk associated with an active.
摘要翻译：提供的组合物包括水溶性或水分散性薄膜碎片，载体和活性剂，例如但不限于药物，调味剂或着色剂。还提供了使用薄膜碎片的方法，例如改善组合物或其组分的溶解，稳定性，美观性或味道，以及降低与活性相关的疾病风险。

7. 发明申请

WO2008100375A3 POLYMER-BASED FILMS AND DRUG DELIVERY SYSTEMS MADE THEREFROM 审中-公开
公开(公告)号：WO2008100375A3
公开(公告)日：2008-08-21
申请号：PCT/US2008/000973
申请日：2008-01-25
申请人： MONOSOL RX LLC , FUISZ, Richard, C. , FUISZ, Joseph, M. , MYERS, Garry, L. , YANG, Robert, K. , SANGHVI, Pradeep, P.
发明人： FUISZ, Richard, C. , FUISZ, Joseph, M. , MYERS, Garry, L. , YANG, Robert, K. , SANGHVI, Pradeep, P.
IPC分类号： A61K9/70
摘要： The invention relates to the film products and methods of their preparation that demonstrate a non-self-aggregating uniform heterogeneity. Desirably, the films disintegrate in water and may be formed by a controlled drying process, or other process that maintains the required uniformity of the film. The films contain a polymer component which includes polyethylene oxide, optionally blended with at least one additional polymer. The films may alternatively contain a polymer component which includes polyvinyl alcohol, optionally blended with at least one additional polymer. The films also may contain a pharmaceutical and/or cosmetic active agent.

8. 发明申请

WO2008066610A1 BALLOON GEOMETRY FOR DELIVERY AND DEPLOYMENT OF SHAPE MEMORY POLYMER STENT WITH FLARES 审中-公开
标题翻译：用于交付和部署形状记忆聚合物柱的气球几何
公开(公告)号：WO2008066610A1
公开(公告)日：2008-06-05
申请号：PCT/US2007/022062
申请日：2007-10-16
申请人： BOSTON SCIENTIFIC SCIMED, INC.
发明人： JORDAN, Gary
IPC分类号： A61M25/10 , A61F2/06 , A61F2/84 , A61L31/14
CPC分类号： A61M25/1002 , A61F2/88 , A61F2/958 , A61F2250/0039 , A61L31/04 , A61L31/14 , A61L2400/16 , A61M2025/1086
摘要： A balloon geometry is utilized herein where the balloon (10) is inflatable from an initial unexpanded state to an expanded state. The balloon includes first (26) and second portions (28); the first portion having a first diameter with the balloon being in the expanded state, the second portion having a second diameter with the balloon being in the expanded state, the first diameter being different from the second diameter. With a stent (12) being mounted onto the balloon, expansion of the balloon results in the first portion of the balloon assisting a first portion of the stent to radially expand more than a second portion' of th' e stent located adjacent to the second portion of the balloon. Preferably, the stent is formed of shape memory polymer.
摘要翻译：这里使用气球几何形状，其中球囊（10）可以从初始未膨胀状态充气至膨胀状态。气囊包括第一部分（26）和第二部分（28）; 所述第一部分具有第一直径，所述球囊处于展开状态，所述第二部分具有第二直径，所述球囊处于展开状态，所述第一直径不同于所述第二直径。通过将支架（12）安装到球囊上，球囊的膨胀导致球囊的第一部分帮助支架的第一部分径向扩张位于第二部分附近的第二部分气球的一部分优选地，支架由形状记忆聚合物形成。

9. 发明申请

WO2008036299A3 EDIBLE WATER-SOLUBLE FILM CONTAINING A FOAM REDUCING FLAVORING AGENT 审中-公开
公开(公告)号：WO2008036299A3
公开(公告)日：2008-03-27
申请号：PCT/US2007/020271
申请日：2007-09-19
申请人： MONOSOL RX LLC , FUISZ, Richard, C. , FUISZ, Joseph, M. , MYERS, Garry, L.
发明人： FUISZ, Richard, C. , FUISZ, Joseph, M. , MYERS, Garry, L.
IPC分类号： A61K9/00 , A61K47/30 , A61K47/36 , A61K47/38 , A61K9/68
摘要： An edible water-soluble film is provided. The film includes at least one water-soluble polymer, and a foam reducing flavoring agent, wherein the film is free of added anti-foaming or defoaming agents.

10. 发明申请

WO2007111851A1 USE OF ADDITIVES TO LOWER THE RATE OF A BINDING REACTION 审中-公开
标题翻译：使用添加剂降低结合反应的速率
公开(公告)号：WO2007111851A1
公开(公告)日：2007-10-04
申请号：PCT/US2007/006681
申请日：2007-03-16
申请人： AOKIN AG , LEVISON, Stuart , NIX, Paul , LEVISON, Derek
发明人： LEVISON, Stuart , NIX, Paul , LEVISON, Derek
IPC分类号： G01N33/53 , G01N33/557
CPC分类号： G01N33/557 , G01N33/5306
摘要： A method of lowering the rate of a specific binding reaction in an assay for the detection and/or measurement of an analyte of interest is provided herein. In particular, the method includes providing a fluorescent conjugate of the analyte; a component capable of specifically binding to the analyte and its fluorescent conjugate; and a sample, which includes or is suspected to include the analyte. The method also includes allowing the specific binding component to interact simultaneously or at different times with the fluorescent conjugate of the analyte and the analyte in the sample, thereby forming a detectable complex due to the reaction between the fluorescent conjugate of the analyte and its specific binding component, wherein the reaction is performed in the presence of non-physiological amounts of at least one additive. The method further includes monitoring for the rate of change of the concentration of the detectable complex as a function of the amount of analyte in the sample.
摘要翻译：本文提供了在用于检测和/或测量感兴趣分析物的测定中降低特异性结合反应速率的方法。特别地，该方法包括提供分析物的荧光共轭物; 能够特异性结合分析物及其荧光缀合物的成分; 以及包含或怀疑包括分析物的样品。该方法还包括允许特异性结合组分与样品中分析物和分析物的荧光缀合物同时或不同时间相互作用，从而由于分析物的荧光偶联物与其特异性结合之间的反应而形成可检测的复合物组分，其中反应在非生理量的至少一种添加剂的存在下进行。该方法还包括根据样品中分析物的量来监测可检测复合物的浓度变化率。

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