Development of gene therapy using genome editing technologies

Abstract

Today, gene therapy using gene editing tool (genome editing therapy) is becoming potential new modality treatment. But we need to consider it comes with unconventional safety issues. AMED research group and taskforce established in Japan Pharmaceutical Manufacturers Association have been cooperating in searching of genome editing therapy products and establishing a safety guideline for genome editing therapy. In this session, we will overview the landscape of genome editing therapy products development as the outcome of this co-working activity.