US 12,064,484 B2
Compositions for introducing RNA into cells
Christian Dohmen, Munich (DE); Christian Plank, Seefeld (DE); Carsten Rudolph, Krailing (DE); and Christian Koch, Munich (DE)
Assigned to ethris GmbH, Planegg (DE)
Appl. No. 14/901,467
Filed by ETHRIS GMBH, Planegg (DE)
PCT Filed Jun. 27, 2014, PCT No. PCT/EP2014/063756
§ 371(c)(1), (2) Date Dec. 28, 2015,
PCT Pub. No. WO2014/207231, PCT Pub. Date Dec. 31, 2014.
Claims priority of application No. 13174390 (EP), filed on Jun. 28, 2013; and application No. 13181380 (EP), filed on Aug. 22, 2013.
Prior Publication US 2017/0021036 A1, Jan. 26, 2017
Int. Cl. A61K 47/00 (2006.01); A61K 9/19 (2006.01); A61K 31/7088 (2006.01); A61K 31/713 (2006.01); A61K 47/26 (2006.01); A61K 47/58 (2017.01); A61K 47/59 (2017.01); A61K 48/00 (2006.01); C08F 8/32 (2006.01); C08G 73/02 (2006.01); C12N 15/87 (2006.01)
CPC A61K 48/0041 (2013.01) [A61K 9/19 (2013.01); A61K 31/713 (2013.01); A61K 47/26 (2013.01); A61K 47/58 (2017.08); A61K 47/59 (2017.08); C08F 8/32 (2013.01); C08G 73/0206 (2013.01); C08G 73/0213 (2013.01); C12N 15/87 (2013.01)] 23 Claims
 
1. A composition comprising a single stranded mRNA and a
a lipidoid having the structure of formula (IV):

OG Complex Work Unit Chemistry
wherein the variables a, b, p, m, n and R1 to R6 are defined as follows:
a is 1 and b is an integer of 2 to 4, or a is an integer of 2 to 4 and b is 1;
p is 1 or 2;
m is 1 and n is 1, m is 2 and n is 0, or m is 2 and n is 1; and
R1 to R6 are independently of each other selected from: hydrogen; —CH2—CH(OH)—R7, —CH(R7)—CH2—OH, —CH2—CH2—(C═O)—NH—R7 and —CH2—R7, wherein R7 is selected from C3-C18 alkyl and C3-C18 alkenyl having one C—C double bond; a protecting group for an amino group; —C(NH)—NH2; a poly(ethylene glycol) chain; and a receptor ligand; provided that at least two of R1 to R6 are —CH2—CH(OH)—R7, —CH(R7)—CH2—OH, —CH2—CH2—(C═O)—NH—R7 or —CH2—R7 and one of R1, R2, R5, and R6 is hydrogen, wherein R7 is selected from C3-C18 alkyl and C3-C18 alkenyl having one C—C double bond; and wherein one or more nitrogen atoms may be protonated.