Patent ID: 11938191
Assignee: GENEVANT SCIENCES GMBH
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 6:
7. A method for the intracellular delivery of an oligonucleotide comprising:
a) contacting a cell with a block copolymer of the formula I

T1-L1-[A]x-[B]y—Z  I

wherein
T1 is a first targeting moiety;
L1 is absent or a linking moiety;
A is a first block that is a polymer formed from monomers comprising formula A2 or a random copolymer formed from monomers comprising formulae A2 and A3; A2, A4 and A5; A2 and A5; or A4 and A5;, wherein n is 1-120, R3 is H or C1-C6 alkyl, R4 is S, O, NH or N(C1-C6 alkyl), R5 is O or S and R6 is H, C1-C6 alkyl, C1-C6 alkyl-NH2, C1-C6 alkyl-NH(C1-C6 alkyl), or C1-C6 alkyl-N(C1-C6 alkyl)2;, wherein R7 and R10 are independently H or C1-C6 alkyl, R8 is S, O, NH or N(C1-C6 alkyl), and R9 is O or S and R11 is an amine protecting group;, wherein n is 1-230, R17 is H or C1-C6 alkyl, R18 is O, S, NH or N(C1-C6 alkyl), R19 is O or S, and R20 is OH, NH, H, T2, or C1-C6 alkyl, where T2 is a second targeting moiety;, wherein R21 is H or C1-C6 alkyl, R22 is O, NH or N(C1-C6 alkyl), R23 is H, aryl, arylhalide, alkyl, or alkyl alcohol;

B is a second block that is a random copolymer formed from monomers consisting of formulae B1, B2, and B3, wherein R12, R13, R14, R15, and R16 are independently H or C1-C6 alkyl,

x is 2-20 kDa;
y is 2-20 kDa;
the ratio of x to y is from 2:1 to 1:4; and
Z is H, SH, C(CH3)2CN,, , wherein R24 is S—(C1-C12 alkyl), aryl, arylhalide, O—(C1-C12 alkyl), or NR25R26 wherein R25 and R26 are independently H, alkyl, aryl, or heteroaryl; and  designates a point of attachment; with a cell and
b) contacting the cell with the oligonucleotide, wherein the oligonucleotide is delivered to the cytosol of the cell.