DISULPHIDE-LINKED POLYETHYLENE GLYCOL/PEPTIDE CONJUGATES FOR NUCLEIC ACID TRANSFECTION Russian patent published in 2015 - IPC A61K47/48 A61P43/00 

FIELD: chemistry.

SUBSTANCE: invention relates to versions of a polymeric carrier molecule, a complex of a polymeric carrier with a load, a method of producing a polymeric carrier molecule, a pharmaceutical composition and a vaccine. In one of the versions, the polymeric carrier molecule has the following formula (I): L-P¹-S-[S-P²-S]_n-S-P³-L and additionally contains in the structure at least one amino acid component (AA)_x, wherein AA is an amino acid, x is an integer selected in the range of 1 to 100. If the amino acid component (AA)_x is present in the structure, then it is a linker between P¹ or P³ and component L. If component L is absent, then at least one amino acid component (AA)_x is part of P¹ or P³. Components P¹ and P³ are different or identical and are a linear or branched chain of a hydrophilic polymer of polyethylene glycol (PEG); P² is a cationic or polycationic peptide or a protein with length of 3 to 100 amino acids, or a cationic or polycationic polymer with molecular weight of 0.5 kDa to 30 kDa; -S-S is a disulphide bond; L is an optional ligand, n denotes an integer selected from 1 to 50. According to the second version, the polymeric carrier molecule has the formula (Ia) l-P¹-S-{[S-P²-S]_a[S-(AA)_X-S]_b}-S-P³-L, where a+b=n, where n equals 1, 2, 3, 4 or 5 to 10; a is an integer selected independent of integer b in the range of 1 to 50, b is an integer selected independent of integer a in the range of 1 to 50. Separate components [S-P²-S] and [S-(AA)_x-S] are present in any order in the subformula {[S-P²-S]_a[S-(AA)_x-S]_b}. The complex of the polymeric carrier with a load is formed by the polymeric carrier molecule and a nucleic acid. A pharmaceutical composition and a vaccine include the complex of the polymeric carrier with a load and optionally a pharmaceutically acceptable carrier and/or solvent. Polymeric carrier molecules and the complex of the polymeric carrier with a load are used as a medicinal agent for treating various diseases.

EFFECT: invention enables to obtain a stable polymeric carrier which enables efficient transfection of nucleic acids in cells in vivo and in vitro.

20 cl, 16 dwg, 2 tbl, 4 ex