ANTISENSE OLIGONUCLEOTIDES OF ANDROGEN RECEPTOR Russian patent published in 2021 - IPC C07K14/00 C12N15/113 A61K38/10 A61K38/16 A61P17/14 

FIELD: biotechnology; medicine.

SUBSTANCE: group of inventions relates to biotechnology and medicine, in particular to derivatives of peptide-nucleic acids (PNA) that interact directionally with pre-mRNA of an androgen receptor. A derivative of PNA of the formula I or its pharmaceutically acceptable salt for the treatment of androgenic alopecia is proposed, where PNA has at least a 9-dimensional complementary site overlapping with a 17-dimensional RNA sequence [(5'→3') CCUUGCCUGGUAAGGAA] in pre-mRNA of the human androgen receptor (AR), and is completely complementary to the pre-mRNA sequence in pre-mRNA of the human AR or partially complementary to the pre-mRNA sequence in pre-mRNA of the human AR with one erroneous pairing. PNA derivatives induce alternative splicing of human pre-mRNA of AR, inhibit the expression of AR, have good cell permeability, and they can be easily delivered to a cell when processed as a “naked” oligonucleotide.

EFFECT: PNA derivatives are applicable for the safe treatment of androgenic alopecia with topical application.

11 cl, 16 dwg, 2 tbl, 11 ex