世界基因治疗药物研发现状与我国发展对策

Global Research and Development Status of Gene Therapy Drug and Development Strategies of Our Country

近年来,随着基因治疗理论的发展和技术的进步,基因治疗药物研发取得了重大突破。我国在基因治疗药物的研发方面基本与发达国家同步,在产业化发展方面抢占了世界先机,具备较强的国际竞争力,但也面临着一些挑战。本文通过对世界基因治疗药物发展现状及其未来研发方向的分析探讨,提出了我国在基因治疗药物的研发过程中,应选择一些病种作为突破口,加强关键科学问题研究,鼓励协同创新与转化,建立健全政策法规和管理体系,促进我国基因治疗领域科技进步和产业有序、健康发展的建议。

In recent years,with the progress of science and technology on gene therapy,the research and development of gene therapy drug has made greats leaps forward. Although China has certain competitiveness on research and development of gene therapy drug,it is still faced with some restrictive factors. By analysis of the current situation and the prospects of future research of gene therapy drug in China and abroad,the paper puts forward some suggestions on the RD of gene therapy drug and its related industries of our country,such as selecting some diseases as breakthrough targets,tackling the key problems,encouraging collaborative innovation and transformation,and improving the policies and regulations and management system.