发明名称 Polyamine derivatives
摘要 Disclosed are compounds, compositions and methods for systemic and local delivery of biologically active molecules.
申请公布号 US9254334(B2) 申请公布日期 2016.02.09
申请号 US201414575555 申请日期 2014.12.18
申请人 CLSN Laboratories, Inc. 发明人 Slobodkin Gregory;Congo Richard;Matar Majed;Fewell Jason;Anwer Khursheed;Sparks Brian Jeffery
分类号 A61K47/48;A61K31/7088;C07C233/38;C07C235/10;C07C235/60;A61K47/16;A61K9/127;A61K31/713;C07D475/04;C07H15/10;C07K7/23;C07K7/64 主分类号 A61K47/48
代理机构 Sterne, Kessler, Goldstein & Fox P.L.L.C. 代理人 Sterne, Kessler, Goldstein & Fox P.L.L.C.
主权项 1. A method for treating a disease in a mammalian subject, said method comprising administering to the subject a therapeutically effective amount of a formulation comprising a biologically active molecule, a compound of Formula A and a compound of Formula B: wherein n1, n2, and n3 in both Formula A and Formula B are independently 1, 2, 3, or 4; X and X′ in both Formula A and Formula B are independently a bond, oxygen, or nitrogen; R1 and R2 in both Formula A and Formula B are independently C8-C25 hydrocarbon groups optionally containing from 1-4 double or triple bonds; in Formula A, G is hydrogen; and in Formula B, G is a polyoxyalkylene, polyvinylpyrrolidone, polyacrylamide, polydimethylacrylamide, polyvinyl alcohol, dextran, poly (L-glutamic acid), styrene maleic anhydride, poly-N-(2-hydroxypropyl) methacrylamide, or polydivinylether maleic anhydride; and wherein the biologically active molecule is selected from the group consisting of: ribosomal RNA; antisense polynucleotides of RNA or DNA; ribozymes; siRNA; shRNA; miRNA; and polynucleotides of genomic DNA, cDNA, or mRNA that encode for a therapeutically useful protein.
地址 Wilmington DE US