LOW-MOLECULAR CONJUGATES FOR INTRACELLULAR DELIVERY OF NUCLEIC ACIDS Russian patent published in 2016 - IPC C12N15/87 C12N15/11 A61K48/00 A61K31/713 A61K47/18 

FIELD: chemistry.

SUBSTANCE: disclosed are compounds and compositions based thereon, used in medicine, of formulae

and

,

a nucleic acid (II), where Y is -(CH₂)₃-; R¹ is -(C1-6)alkyl; or -(CH₂)_m-phenyl, optionally substituted up to four times with a substitute selected from: -NO₂, -CN, or a halogen; R² is hydrogen; -(CH₂)_k-phenyl; -(C1-6)alkyl; -(CH₂)_k-C(O)-NH₂; or -(CH₂)_k-N-C(Ph)₃. The phenyl rings are optionally substituted with -O-(C1-4)alkyl; R³ is -NH-phenyl, wherein the phenyl is further substituted with a substitute independently selected from -(CH₂)-OH or -(CH₂)-O-C(O)-O-(4-nitrophenyl); k equals 1, 2, 3, 4, 5 or 6; m equals 1, 2, 3 or 4; and n equals 0 or 1, R^a is -(CH₂)_k-NH₂; R¹, wherein the nucleic acid is a single-stranded RNA oligonucleotide.

EFFECT: novel compounds for nucleic acid delivery in cells and compounds for production thereof.

10 cl, 653 ex, 19 tbl, 6 dwg