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1. 发明申请

WO2014179562A8 1,3,5-TRIAZINANE-2,4,6-TRIONE DERIVATIVES AND USES THEREOF 审中-公开
标题翻译： 1,3,5-三亚乙基-2,4,6-三酮衍生物及其用途
公开(公告)号：WO2014179562A8
公开(公告)日：2015-01-08
申请号：PCT/US2014036355
申请日：2014-05-01
申请人： MASSACHUSETTS INST TECHNOLOGY
发明人： DONG YIZHOU , ALABI AKINLEYE C , YIN HAO , DORKIN JOSEPH R , CHEN DELAI , LANGER ROBERT S , ANDERSON DANIEL GRIFFITH , ELTOUKHY AHMED A
IPC分类号： C07D251/30
CPC分类号： C07D251/30 , A61K31/53 , A61K31/713
摘要： The present invention provides novel l,3,5-triazinane-2,4,6-trione derivatives, such as compounds of any one of Formulae (I) and (II), and salts thereof, and methods of preparing the compounds. Also provided are compositions including a compound of the invention and an agent (e.g., an siRNA, mRNA, or plasmid DNA). The present invention also provides methods and kits using the compositions for delivering an agent to a subject (e.g., to the liver, spleen, or lung of the subject) or cell and for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, and lung diseases.
摘要翻译：本发明提供了新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物，例如式（I）和（II）中任一项的化合物及其盐，以及制备该化合物的方法。还提供了包含本发明化合物和试剂（例如siRNA，mRNA或质粒DNA）的组合物。本发明还提供了使用组合物将试剂递送至受试者（例如，受试者的肝脏，脾脏或肺）或细胞的方法和试剂盒，并用于治疗和/或预防一系列疾病，例如遗传疾病，增殖性疾病，血液疾病，神经系统疾病，肝脏疾病和肺部疾病。

2. 发明申请

WO2015191693A3 METHOD FOR GENE EDITING 审中-公开
标题翻译：基因编辑方法
公开(公告)号：WO2015191693A3
公开(公告)日：2016-02-04
申请号：PCT/US2015035077
申请日：2015-06-10
申请人： MASSACHUSETTS INST TECHNOLOGY
发明人： YIN HAO , XUE WEN , ANDERSON DANIEL G , DORKIN JOSEPH R , JACKS TYLER E
IPC分类号： C12P21/06 , C12N15/10 , C12N15/63
CPC分类号： C12N15/102 , A61K48/005 , C12N7/00 , C12N15/111 , C12N15/85 , C12N15/86 , C12N15/88 , C12N2310/20 , C12N2750/14143
摘要： The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.
摘要翻译：本公开涉及用于修饰基因序列的组合物和方法，以及用于递送该组合物的系统。例如，本公开涉及使用CRISPR-Cas9或其他核酸编辑系统修饰基因序列，以及用于实现这种基因修饰的方法和递送系统，例如病毒或非病毒递送系统。

3. 发明申请

WO2013090861A8 ALPHA-AMINOAMIDINE POLYMERS AND USES THEREOF 审中-公开
标题翻译： α-氨基酰胺聚合物及其用途
公开(公告)号：WO2013090861A8
公开(公告)日：2013-08-22
申请号：PCT/US2012069961
申请日：2012-12-15
申请人： MASSACHUSETTS INST TECHNOLOGY
发明人： VEGAS ARTURO JOSE , WHITEHEAD KATHRYN ANN , ANDERSON DANIEL GRIFFITH , LANGER ROBERT S , DORKIN JOSEPH R
IPC分类号： C08G73/00 , A61K9/127 , A61K9/51 , A61K47/34 , C08L79/00 , C12N15/88
CPC分类号： A61K47/34 , A61K9/1271 , A61K9/5146 , A61K31/713 , A61K33/00 , A61K38/16 , A61K39/00 , C08G12/06 , C08G12/34 , C08G73/00 , C08L79/00 , C12N15/113
摘要： alpha-Aminoamidine polymers and methods of preparing a-aminoamidine polymers by reacting by reacting one or more amines with one or more isocyanides and one or more aldehydes are described. Methods of preparing a-aminoamidine polymers from commercially available starting materials are also provided, wherein the starting materials are racemic or stereochemically pure. a-Aminoamidine polymers or salt forms thereof are preferably biodegradable and biocompatible and may be used in a variety of drug delivery systems and for other purposes as well such as, for example, coatings, additives, excipients, plastics, and materials, etc. Given the amino moiety of these alpha-aminoamidine polymers, they are particularly suited for the delivery of polynucleotides. Complexes, micelles, liposomes or particles containing the inventive alpha-aminoamidine polymers and polynucleotides can be prepared. The inventive alpha-aminoamidine polymers may also be used in preparing microparticles for drug delivery. They are particularly useful in delivering labile agents given their ability to buffer the pH of their surroundings.
摘要翻译： α-氨基脒聚合物以及通过使一种或多种胺与一种或多种异氰化物和一种或多种醛反应来制备α-氨基脒聚合物的方法。还提供了由市售原料制备α-氨基脒聚合物的方法，其中原料是外消旋或立体化学纯的。 α-氨基脒聚合物或其盐形式优选是可生物降解的和生物相容的，并且可以用于多种药物递送系统中以及用于其他目的，例如涂料，添加剂，赋形剂，塑料和材料等。给定的这些α-氨基脒聚合物的氨基部分，它们特别适合于递送多核苷酸。可以制备含有本发明α-氨基脒聚合物和多核苷酸的复合物，胶束，脂质体或颗粒。本发明的α-氨基脒聚合物也可以用于制备用于药物递送的微粒。考虑到它们缓冲周围环境pH的能力，它们在提供不稳定剂方面特别有用。

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