Uchida Eriko, Igarashi Yuka, Sato Yoji
Kokuritsu Iyakuhin Shokuhin Eisei Kenkyusho Hokoku. 2014(132):10-2.
Gene therapy products are expected as innovative medicinal products for intractable diseases such as life-threatening genetic diseases and cancer. Recently, clinical developments by pharmaceutical companies are accelerated in Europe and the United States, and the first gene therapy product in advanced countries was approved for marketing authorization by the European Commission in 2012. On the other hand, more than 40 clinical studies for gene therapy have been completed or ongoing in Japan, most of them are conducted as clinical researches by academic institutes, and few clinical trials have been conducted for approval of gene therapy products. In order to promote the development of gene therapy products, revision of the current guideline and/or preparation of concept paper to address the evaluation of the quality and safety of gene therapy products are necessary and desired to clearly show what data should be submitted before First-in-Human clinical trials of novel gene therapy products. We started collaborative study with academia and regulatory agency to promote regulatory science toward clinical development of gene therapy products for genetic diseases based on lentivirus and adeno-associated virus vectors; National Center for Child Health and Development (NCCHD), Nippon Medical School and PMDA have been joined in the task force. At first, we are preparing pre-draft of the revision of the current gene therapy guidelines in this project.
基因治疗产品有望成为治疗诸如危及生命的遗传性疾病和癌症等疑难病症的创新型药品。近来，欧美制药公司的临床研发进程加快，2012年，发达国家首个基因治疗产品获欧盟委员会批准上市。另一方面，日本已完成或正在进行40多项基因治疗临床研究，其中大部分是学术机构开展的临床研究，为批准基因治疗产品进行的临床试验较少。为推动基因治疗产品的研发，有必要修订现行指南和/或制定概念文件，以评估基因治疗产品的质量和安全性，明确新型基因治疗产品首次人体临床试验前应提交哪些数据。我们与学术界和监管机构展开合作研究，以推动基于慢病毒和腺相关病毒载体的遗传性疾病基因治疗产品临床开发的监管科学；国立儿童健康与发展中心（NCCHD）、日本医科大学和日本药品和医疗器械管理局（PMDA）已加入该特别工作组。首先，我们正在为本项目中现行基因治疗指南的修订准备预草案。
