PBGENE-PCSK9

PBGENE-PCSK9 is an investigational in vivo gene therapy targeting the PCSK9 gene to achieve long-term lowering of low-density lipoprotein cholesterol in patients with hypercholesterolemia, aiming for a “one-and-done” therapeutic effect via permanent reduction of hepatic PCSK9 activity. PCSK9 regulates LDL receptor degradation; silencing or editing PCSK9 increases hepatic LDL receptor recycling and clearance of LDL-C from blood, which has been shown to durably reduce LDL-C in preclinical primate studies of PCSK9-directed gene editing approaches.[2] PCSK9 inhibition or suppression is an established strategy that lowers LDL-C by about 50–60% with current modalities (monoclonal antibodies and siRNA), supporting the therapeutic rationale for gene-based targeting of PCSK9 for a longer-lasting effect.[7][1]

Other names

PCSK9-directed ARCUS nucleasePCSK-9-directed ARCUS nucleasePCSK 9-directed ARCUS nucleaseARCUS nuclease targeting PCSK9