LIPID-BASED FORMULATIONS FOR RNA DELIVERY Russian patent published in 2024 - IPC A61K9/127 A61K31/7105 A61K47/10 A61K47/24 A61K47/28 A61K47/69 A61K48/00 A61P11/00 

FIELD: pharmaceutical industry.

SUBSTANCE: group of inventions relates to a composition for treatment or prevention of a disease using mRNA-based therapy; to the method of obtaining it; to its use in the treatment or prevention of disease using mRNA-based therapy. The proposed composition contains (i) particles in a liquid phase, where the particles include mRNA and a lipid composition, and (ii) 1,2-propanediol, wherein the lipid composition includes: (i-a) a cholesterol derivative of formula (I) or pharmaceutically or physiologically acceptable salt thereof

where n is 0, R¹ means a -(CH₂)_q-NH₂ group or a -(CH₂)_r-NH-(CH₂)_s-NH₂ group, R² means a -(CH₂)_t-NH group₂ or a group -(CH₂)_u-NH-(CH₂)_w-NH₂, in which q, r, s, t, u, w are independently an integer from 2 to 6; (i-b) a phosphoglyceride of formula (II) or a pharmaceutically or physiologically acceptable salt thereof

where R⁴ and R⁵ each means a linear alkenyl group containing 10–24 carbon atoms and from 1 to 3 double bonds; (i-c) a pegylated phosphoglyceride of formula (III) or a pharmaceutically or physiologically acceptable salt thereof

where p is an integer from 5 to 200, R⁶ and R⁷ each represent a linear alkyl group containing 10–20 carbon atoms. The proposed method is characterized by the following steps: a) the components of the lipid composition in an organic solvent are dissolved and mixed, then its lyophilization is performed; b) the lyophilized lipid composition is rehydrated by adding water; c) the rehydrated lipid composition is combined with an aqueous solution of RNA; d) 1,2-propanediol is added.

EFFECT: group of inventions improves the efficiency of transfection of compositions based on mRNA and lipids.

14 cl, 3 dwg, 2 tbl, 3 ex