COMPOSITIONS OF NON-VIRAL NON-CAPSID DNA VECTORS BASED ON LIPID NANOPARTICLES Russian patent published in 2022 - IPC C12N15/86 A61K9/51 A61K9/127 A61K48/00 

Abstract RU 2778407 C2

FIELD: biotechnology.

SUBSTANCE: group of inventions is described, including a lipid nanoparticle for the delivery of a non-capsid non-viral DNA vector to a target site, and a pharmaceutical composition for the delivery of heterologous nucleic acid (options). In one option of the implementation, the lipid nanoparticle contains an ionized lipid and a non-viral non-capsid DNA vector with covalently closed ends (hereinafter – ceDNA vector), where the specified ceDNA vector contains at least one heterologous nucleotide sequence functionally located between asymmetrical inverted terminal repeats (hereinafter – asymmetrical ITR), where ITR are asymmetrical relatively to each other, and where one or more of asymmetrical ITR is modified by deletion, insertion, and/or replacement in at least one of areas selected from A, A’, B, B’, C, C’, D, and D’, wherein at least one of asymmetrical ITR contains a functional site of terminal resolution and Rep binding site.

EFFECT: invention expands the arsenal of means for the delivery of a non-capsid non-viral DNA vector to a target site.

37 cl, 37 dwg, 5 tbl, 13 ex

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RU 2 778 407 C2

Authors

Kotin, Robert Michael

Alkan, Ozan

Kerr, Douglas Anthony

Malakian, Ara Karl

Simmons, Matthew John

Stanton, Matthew G.

Su, Jie

Wright, Teresa L.

Dates

2022-08-18Published

2018-09-07Filed