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    BRUSH-POLY (GLYCOAMIDOAMINE)-LIPIDS AND USES THEREOF
    47.
    发明申请
    BRUSH-POLY (GLYCOAMIDOAMINE)-LIPIDS AND USES THEREOF 审中-公开
    BRUSH-POLY（GLYCOAMIDOAMINE） - 脂类及其用途

    公开(公告)号：US20160067346A1

    公开(公告)日：2016-03-10

    申请号：US14825127

    申请日：2015-08-12

    Applicant: Massachusetts Institute of Technology

    Inventor： Yizhou Dong ,  Joseph R. Dorkin ,  Robert S. Langer ,  Daniel Griffith Anderson

    IPC: A61K47/48 ,  A61K38/18 ,  C12N15/113 ,  A61K9/127 ,  C08G69/48 ,  C08G69/50

    CPC classification number: A61K47/48207 ,  A61K9/1273 ,  A61K38/1816 ,  A61K47/544 ,  A61K47/554 ,  A61K47/595 ,  A61K47/60 ,  A61K47/6915 ,  A61K47/6935 ,  A61K48/00 ,  A61K48/0041 ,  C08G69/48 ,  C08G69/50 ,  C08G73/00 ,  C08L79/00 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/32

    Abstract: The present disclosure provides brush-poly(glycoamidoamine)-lipids (PGALs) (e.g., polymers of any one of Formulae (I)-(IV)) and methods of preparing the PGALs. A described PGAL may include poly(glycoamidoamine)-derived moieties (e.g., such as which may assist the PGAL and/or a complex of the PGAL and an agent to pass through cell membranes or be taken up by cells. Also provided are compositions including a described PGAL and an agent (e.g., polynucleotide, small molecule, peptide, or protein). The present disclosure also provides methods, kits, and uses that include or involve the PGALs or compositions for delivering an agent to a subject, tissue, or cell and/or for treating and/or preventing in a subject a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, immunological diseases, gastrointestinal diseases, respiratory diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, genitourinary diseases, and metabolic disorders.

    Abstract translation: 本公开内容提供了刷 - 聚（糖醇胺）脂质（PGAL）（例如式（I） - （IV）中任一项的聚合物））和制备PGAL的方法。 所描述的PGAL可以包括衍生自聚（糖糖胺基胺）的部分（例如，可以帮助PGAL和/或PGAL和试剂的复合物通过细胞膜或被细胞摄取），还提供包括 所描述的PGAL和药剂（例如，多核苷酸，小分子，肽或蛋白质），本公开还提供了包括或涉及用于将药剂递送至受试者，组织或组织的PGAL或组合物的方法，试剂盒和用途 细胞和/或用于在受试者中治疗和/或预防一系列疾病，例如遗传疾病，增殖性疾病，血液病，神经疾病，免疫疾病，胃肠道疾病，呼吸系统疾病，疼痛病症，精神病学，肌肉骨骼疾病， 泌尿生殖系疾病和代谢紊乱。

    BIODEGRADABLE POLY(BETA-AMINO ESTERS) AND USES THEREOF
    49.
    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：US20140094399A1

    公开(公告)日：2014-04-03

    申请号：US14029552

    申请日：2013-09-17

    Applicant: Massachusetts Institute of Technology

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

    IPC: A61K47/34

    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。

    IONIZABLE LIPIDS FOR MULTIPLE ORGAN TARGETING
    50.
    发明申请
    IONIZABLE LIPIDS FOR MULTIPLE ORGAN TARGETING 有权

    公开(公告)号：US20250064938A1

    公开(公告)日：2025-02-27

    申请号：US18725289

    申请日：2023-01-03

    Applicant: Massachusetts Institute of Technology

    Inventor： Daniel Griffith Anderson ,  Bowen Li ,  Akiva Gordon ,  Idris Raji

    IPC: A61K47/54 ,  A61K9/127 ,  A61K31/7105 ,  A61K48/00 ,  C07C233/47 ,  C07D211/14 ,  C07D231/12 ,  C07D233/61 ,  C07D243/08 ,  C07D295/13 ,  C12N15/113

    Abstract: Provided herein are lipid compounds of Formulae (I) and (II), and pharmaceutically acceptable salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits involving the inventive lipid compounds, compositions, or formulations for treating and/or preventing diseases (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, painful condition, psychiatric disorder, metabolic disorder, long-term medical condition, inflammatory disease, autoinflammatory disease, liver disease, lung disease, spleen disease, familial amyloid neuropathy, cardiovascular disease, viral infection, infectious disease, fibrotic condition, or autoimmune disease) in a subject, methods for synthesizing the compounds described herein, and compounds described herein synthesized by the synthetic methods described herein. The compounds are effective carriers for the delivery of an agent such as a polynucleotide (e.g., RNA) to a tissue or cell in a subject (e.g., a liver, lung, or spleen tissue/cell).

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