Patent ID: 6468986
Filing Date: 2002-10-22
Classification: A61K,A61P,C07K,C12N

Abstract:
A composition comprising:(i) a polynucleotide; and (ii) a polycationic agent having the following formula: whereinn is an integer from 10 to 100 resulting in a repeating trimer wherein two R1 groups of each trimer are neutral and one R1 group of each trimer is cationic; R2 and R3 are hydrogen for each monomer: Ta and Tc are terminating groups; said cationic R1 groups are selected from the group consisting of ammoniumalkyl, aminoalkyl, guanidinoalkyl, amidinoalkyl, aminocyclohexyl, piperidyl, guanidinobenzyl, guanidino-&agr;-methylbenzyl, amidinobenzyl, amidino-&agr;-methylbenzyl, an aliphatic hydrazide, an aromatic hydrazide; pyridylmethyl, wherein the pyridyl ring may be further substituted with methyl, methoxy, fluoro, chloro, bromo, or nitro; and aminobenzyl, wherein the benzyl ring may be further substituted with methyl, methoxy, fluoro, chloro, bromo, or nitro; wherein â€œammoniumâ€  is trimethylammonium, triethylammonium, methyldiethylammonium, or ethyldimethylammonium, and â€œalkylâ€  is â€”CH(R) â€”(CH2)m-, where R is H or Me, and m is 1-6; and said neutral R1 groups are selected from the group consisting of a neutral moiety comprising an aromatic ring and having a molecular weight of 250 daltons or less, cyclohexyl methyl, 2-methoxyethyl, and 6-galactosyl; wherein said polycationic agent comprises at least 25% positively charged monomers, excluding the terminal groups, and wherein said polycationic agent exhibits a net positive electrical charge at a physiological pH, and is capable of mediating entry of polynucleotides into a cell.