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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
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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公开(公告)号：US09840479B2
公开(公告)日：2017-12-12
申请号：US14789227
申请日：2015-07-01
Applicant: Massachusetts Institute of Technology
IPC: A61K31/495 , C07D241/08 , C07C219/06 , C07C219/08 , C07D295/088
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.
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