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    MULTI-TAILED LIPIDS AND USES THEREOF
    26.
    发明申请
    MULTI-TAILED LIPIDS AND USES THEREOF 有权
    多用途脂肪及其用途

    公开(公告)号：US20160158363A1

    公开(公告)日：2016-06-09

    申请号：US14900869

    申请日：2014-06-26

    Applicant: MASSACHUSETTS INSTITUTE OF TECHNOLOGY

    Inventor： Akinleye C. Alabi ,  Kevin Thomas Love ,  Daniel Griffith Anderson ,  Robert S. Langer

    IPC: A61K47/22 ,  C07D207/09 ,  C12N15/113 ,  C07D295/15

    CPC classification number: A61K47/22 ,  A61K9/127 ,  C07C323/12 ,  C07D207/09 ,  C07D295/15 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/32

    Abstract: The present invention provides multi-tailed lipid compounds, and salts and stereoisomers 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, plasmid DNA, small molecule, protein, peptide). 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, immunological diseases, gastrointestinal diseases (e.g., liver diseases), respiratory diseases (e.g., lung diseases), painful conditions, psychiatric disorders, metabolic disorders, and spleen diseases.

    Abstract translation: 本发明提供多尾脂质化合物及其盐和立体异构体，以及制备化合物的方法。 还提供了包含本发明化合物和试剂（例如siRNA，mRNA，质粒DNA，小分子，蛋白质，肽）的组合物。 本发明还提供了使用组合物将试剂递送到受试者（例如，受试者的肝脏，脾脏或肺部）或细胞并用于治疗和/或预防一系列疾病的方法和试剂盒，例如 遗传疾病，增殖性疾病，血液病，神经系统疾病，免疫疾病，胃肠道疾病（如肝脏疾病），呼吸系统疾病（如肺部疾病），疼痛病症，精神疾病，代谢疾病和脾脏疾病等。

    1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
    27.
    发明授权
    1,3,5-triazinane-2,4,6-trione derivatives and uses thereof 有权
    1,3,5-三氮杂环戊烷-2,4,6-三酮衍生物及其用途

    公开(公告)号：US09315472B2

    公开(公告)日：2016-04-19

    申请号：US14267530

    申请日：2014-05-01

    Applicant: Massachusetts Institute of Technology

    Inventor： Yizhou Dong ,  Akinleye C. Alabi ,  Hao Yin ,  Joseph R. Dorkin ,  Delai Chen ,  Robert S. Langer ,  Daniel Griffith Anderson ,  Ahmed A. Eltoukhy

    IPC: C07D251/30 ,  A61K31/53 ,  A61K31/713

    CPC classification number: C07D251/30 ,  A61K31/53 ,  A61K31/713

    Abstract: The present invention provides novel 1,3,5-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.

    Abstract translation: 本发明提供新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物，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。

    BIODEGRADABLE POLY (BETA-AMINO ESTERS) AND USES THEREOF
    28.
    发明申请
    BIODEGRADABLE POLY (BETA-AMINO ESTERS) AND USES THEREOF 有权
    生物可降解聚（氨基甲酸酯）及其用途

    公开(公告)号：US20160022821A1

    公开(公告)日：2016-01-28

    申请号：US14811613

    申请日：2015-07-28

    Applicant: Massachusetts Institute of Technology

    Inventor： Robert S. Langer ,  David M. Lynn ,  David A. Putnam ,  Mansoor M. Amiji ,  Daniel Griffith Anderson

    IPC: A61K47/34 ,  A61K31/711 ,  A61K31/713 ,  C08G63/685

    CPC classification number: A61K47/34 ,  A61K9/5146 ,  A61K31/711 ,  A61K31/713 ,  A61K47/59 ,  A61K47/593 ,  A61K47/6929 ,  B82Y5/00 ,  C08G63/685 ,  C08G73/02 ,  C08G73/0273 ,  C08G73/06 ,  C08G73/0611 ,  C08G73/0616 ,  C12N15/88

    Abstract: Poly(β-amino esters) prepared from the conjugate addition of bis(secondary amines) or primary amines to a bis(acrylate ester) are described. Methods of preparing these polymers from commercially available starting materials are also provided. These tertiary amine-containing polymers are preferably biodegradable and biocompatible and may be used in a variety of drug delivery systems. Given the poly(amine) nature of these polymers, they are particularly suited for the delivery of polynucleotides. Nanoparticles containing polymer/polynucleotide complexes have been prepared. The inventive polymers may also be used to encapsulate other agents to be delivered. They are particularly useful in delivering labile agents given their ability to buffer the pH of their surroundings.

    Abstract translation: 描述了从双（仲胺）或伯胺向双（丙烯酸酯）共轭加成制备的聚（＆（bgr。 - 氨基酯））。 还提供了从市售的原料制备这些聚合物的方法。 这些含叔胺的聚合物优选是可生物降解的和生物相容的并且可以用于各种药物递送系统中。 鉴于这些聚合物的聚（胺）性质，它们特别适用于递送多核苷酸。 已经制备了含有聚合物/多核苷酸复合物的纳米颗粒。 本发明的聚合物也可以用于包封待递送的其它试剂。 考虑到缓冲其周围环境的pH值，它们特别适用于提供不稳定剂。

    POLYAMINE-FATTY ACID DERIVED LIPIDOIDS AND USES THEREOF
    29.
    发明申请
    POLYAMINE-FATTY ACID DERIVED LIPIDOIDS AND USES THEREOF 有权
    多胺脂肪酸衍生的脂蛋白及其用途

    公开(公告)号：US20160002178A1

    公开(公告)日：2016-01-07

    申请号：US14789227

    申请日：2015-07-01

    Applicant: Massachusetts Institute of Technology

    Inventor： Owen Shea Fenton ,  Joseph Robert Dorkin ,  Daniel Griffith Anderson ,  Rebecca L. McClellan

    IPC: C07D241/08

    CPC classification number: C07D241/08 ,  C07C219/06 ,  C07C219/08 ,  C07D295/088

    Abstract: The present disclosure provides polyamine-fatty acid derived lipidoids (e.g., compounds of Formula (I) or (II)) and methods of preparing the lipidoids. A described lipidoid includes R—C(═O)—O— moieties (where R is a lipid moiety), which may be hydrolyzed into non-toxic fatty acids. Also provided are compositions including a described lipidoid and an agent (e.g., polynucleotide, small molecule, peptide, or protein). The present disclosure also provides methods, kits, and uses that involve the lipidoids or compositions for delivering an agent to a subject, tissue, or cell and/or for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, immunological diseases, gastrointestinal diseases, respiratory diseases, painful conditions, psychiatric disorders, and metabolic disorders.

    Abstract translation: 本公开提供多胺 - 脂肪酸衍生的类固醇（例如式（I）或（II）的化合物）和制备类固醇的方法。 所描述的脂质包括可以被水解成无毒性脂肪酸的R-C（= O）-O-部分（其中R是脂质部分）。 还提供了包含所述类脂质和试剂（例如多核苷酸，小分子，肽或蛋白质）的组合物。 本公开还提供涉及将类固醇或组合物递送到受试者，组织或细胞和/或用于治疗和/或预防一系列疾病如遗传疾病，增殖性疾病的方法，试剂盒和用途 血液疾病，神经系统疾病，免疫疾病，胃肠道疾病，呼吸系统疾病，疼痛病症，精神疾病和代谢性疾病。

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