Current gene therapy for hemophilia and the Cartagena Act

Abstract

The quality of life of hemophilia patients has considerably been improved because unexpected bleeding has become preventable by prophylactic treatments with coagulation factor concentrates. The frequency of injections has decreased along with the development of extended half-life coagulation products and biphasic antibody preparations. However, pain and mental burden for regular replacement therapy remain as problems. The progress of gene and cell therapies is remarkable for many intractable diseases. In gene therapy for hemophilia, worldwide competition in research and development of new therapies has led to clinical trials in Japan. Adenovirus-associated viral vectors are intravenously injected to hemophilia patients. Single administration of the vectors with high transfer efficiency into hepatocytes achieves the production of coagulation factors over a long time. Development of gene and cell therapies for pediatric diseases is being aspired for; however, the therapies are still in developmental stages. We need new evaluation methods for safety and efficacy that are different from those for conventional medicines. For example, viral preparations necessitate a special implementation system at hospitals in accordance with the Cartagena Act. Moreover, when using viral preparations for treatment, it is necessary to establish a special implementation system in medical facilities in accordance with the Cartagena Act. Considering these requirements, we launched the Gene Cell Therapy Promotion Center in 2019 to provide a concrete basis of clinical trials and long-term follow-up. The Center aims at supporting companies and academia to prepare clinical trial protocols and education/training. In this paper, we outline the current status of gene therapy for hemophilia and its conformity with the Cartagena Act.