公开(公告)号：US20160114042A1

公开(公告)日：2016-04-28

申请号：US14987717

申请日：2016-01-04

Applicant: Massachusetts Institute of Technology

Inventor： Daniel Griffith Anderson ,  Kathryn Ann Whitehead ,  Joseph R. Dorkin ,  Arturo Jose Vegas ,  Yunlong Zhang ,  Robert S. Langer

IPC: A61K47/22 ,  C07D241/04 ,  C12N15/113 ,  C07D295/13

CPC classification number: A61K47/22 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/225 ,  A61K31/357 ,  A61K31/40 ,  A61K31/445 ,  A61K31/495 ,  A61K31/4985 ,  A61K31/573 ,  A61K31/704 ,  A61K31/713 ,  A61K45/06 ,  A61K47/18 ,  A61K47/542 ,  A61K47/543 ,  A61K47/6925 ,  C07C225/06 ,  C07C229/12 ,  C07C229/14 ,  C07C229/24 ,  C07C229/26 ,  C07C229/28 ,  C07C229/34 ,  C07C2601/14 ,  C07D207/09 ,  C07D211/14 ,  C07D211/26 ,  C07D211/34 ,  C07D241/04 ,  C07D295/13 ,  C07D295/15 ,  C07D295/16 ,  C07D319/16 ,  C07D319/18 ,  C07D487/04 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/88 ,  C12N2310/14 ,  C12N2320/32 ,  A61K2300/00

Abstract: Provided herein are lipidoids that may be prepared from the conjugate addition of alkylamines to acrylates. In some embodiments, provided lipidoids are biodegradable and may be used in a variety of drug delivery systems. Given the amino moiety of the lipidoids, they are well-suited for the delivery of polynucleotides, in addition to other agents. Nanoparticles containing the inventive lipidoids and polynucleotides have been prepared and have been shown to be effective in delivering siRNA.

Abstract translation: 本文提供的是可以通过将烷基胺偶联加成丙烯酸酯制备的类脂。 在一些实施方案中，提供的类脂质是可生物降解的并且可以用于多种药物递送系统中。 考虑到类固醇的氨基部分，除了其它药剂之外，它们非常适合于递送多核苷酸。 已经制备了含有本发明的类脂和多核苷酸的纳米颗粒，并且已显示其在递送siRNA方面是有效的。

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。