知识图谱视角下的细胞基因治疗关键技术与产业发展  

Cell and Gene Therapy Key Technology and Industry Development from the Perspective of Knowledge Graph

作　　者：李荣[1] 范月蕾[1] 张博文[1] 毛开云[1] LI Rong;FAN Yuelei;ZHANG Bowen;MAO Kaiyun(Shanghai Information Center for Life Sciences,Shanghai Institute of Nutrition and Health,Chinese Academy of Sciences,Shanghai 200031,China)

机构地区：[1]中国科学院上海生命科学信息中心,中国科学院上海营养与健康研究所,上海200031

出　　处：《竞争情报》2024年第5期19-27,共9页Competitive Intelligence

基　　金：2024年苏州市软科学研究重点项目“苏州市合成生物及基因与细胞治疗技术发展研究”的研究成果。

摘　　要：细胞基因治疗(Cell and Gene Therapy,CGT)指将遗传物质或者细胞转移至患者体内,用于疾病治疗的过程,作为一种先进的生物治疗手段,其所涉及的技术众多、产品分类广泛、临床应用前景广阔,是当前备受关注的领域。系统梳理CGT产业上游所涉及的关键技术与所用设备耗材,产业中游的药物制备,产业下游的市场终端与应用场景,产业配套服务的合同研究组织(Contract Research Organization,CRO)、合同研发生产组织(Contract Development and Manufacturing Organization,CDMO)与检验检测,根据重要技术之间的关联性和逻辑关系,绘制出CGT产业知识图谱。基于知识图谱,分析靶点开发、基因递送策略、新型基因编辑工具、嵌合抗原受体(Chimeric Antigen Receptor,CAR)分子结构设计等关键技术,进一步分析了嵌合抗原受体T细胞免疫(Chimeric Antigen Receptor T-Cell immunotherapy,CAR-T)疗法、诱导多功能干细胞(Induced Pluripotent Stem Cell,iPSC)疗法和核酸类药物的开发进展,并分析CRO和CDMO对于提升CGT产业的发展效率。可以预见,CGT产业将会涌现和开发更多的技术,药物上市进程将会更快。Cell and gene therapy(CGT)refers to the proc ess of transferring genetic material or cells into the patient’s body for disease treatment.As an advanced treatment,it involves a wide range of technologies and product categories,and broad clinical application.This article systematically reviews the key technologies and equipment and consumables involved in the upstream of the CGT industry,the drug production in the midstream of the industry,the market terminals and application scenarios in the downstream of the industry,and the contract research organization(CRO),contract development and manufacturing organization(CDMO)and inspection and test in the industry.According to the correlation and logical relationship between important technologies,we draw the CGT industry knowledge graph.Based on the knowledge graph,we analyzed key technologies such as target development,gene delivery strategies,new gene editing tools,and CAR molecular structure design.We further analyzed the development progress of chimeric antigen receptor T-cell immunotherapy(CAR-T therapy),induced pluripotent stem cell(iPSC)therapy and nucleic acid drugs,and analyzed the function of CRO and CDMO in improving the development efficiency of the CGT industry.It can be foreseen that more technologies will be developed in the CGT industry,and the process of drug launch will be faster.

关 键 词：知识图谱 细胞基因治疗 关键技术 产业 

分 类 号：R450[医药卫生—治疗学] G353.1[医药卫生—临床医学]