This invention discloses a
circular RNA agent targeting PCSK9 and its applications, relating to the field of
biomedical technology. The
circular RNA agent is a single-stranded closed-circular
oligonucleotide containing an antisense sequence complementary to the transcription of the PCSK9
gene. It lacks free 5' and 3' ends, and does not contain overhangs, hairpin structures, or double-stranded regions. The molecules are linked by 3'-5' phosphodiester bonds. This
circular RNA agent can be modified with GalNAc or prepared into pharmaceutical compositions for the treatment of diseases or conditions related to abnormal PCSK9
gene expression or activity. The circular
RNA agent provided by this invention exhibits high stability, a long half-life, low
immunogenicity, low modification
toxicity, and high
target gene inhibitory activity. It can significantly prolong the
dosing interval, improve
patient compliance, and provide a
safer, longer-acting
drug option for lipid-lowering therapy.