Abstract

First Commercial Available Gene Therapy Drug “Collategen” in Cardiovascular Disease

Ryuichi Morishita, Professor of Clinical Gene Therapy, Osaka University School of Medicine

Gene therapy has emerged as a novel therapy to promote angiogenesis in patients with critical limb ischemia (CLI) caused by peripheral artery disease. We have focused on hepatocyte growth factor (HGF) as pro-angiogenic factors. In the clinical trials of phase III, the naked plasmid DNA encoding HGF showed the safety and their potential for symptomatic improvement in CLI patients. Improvement rate was 70.4 % in HGF and 30.8% in placebo in Fontaine III patients (P=0.014). HGF gene therapy achieved a significantly higher improvement rate of ischemic ulcers compared with placebo (100% vs. 40%, P=0.018). No major safety problems were observed. Based on phase III data, in this year, HGF will be launched in Japan market as the first gene therapy drug.