Stem Cells with Gene Editing

Gene editing is a novel gene engineeering tool that precisely modify target gene sequence. The mainly approaches of gene editing in basic and translational medicines include CRISPR/Cas9, single base editing (ABE/CBE), prime editing.The gene editing is succesfully employed in clinical trials of treatment of β-thalessemia and Duchenne muscular dystrophy.

Our company aims to develope gene editing medicines for hyperlipemia, as well as various genetic diseases.

PCSK9 is a hot target for hypercholesterolemia. The current medicine in markets mostly are antibody or SiRNA for PCSK9.

Our company is developing RNA editing tools for PCSK9 to treat hypercholesterolemia.