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中国医药导刊 ›› 2023, Vol. 25 ›› Issue (1): 13-20.

• 专栏—基因治疗 • 上一篇    下一篇

细胞与基因治疗行业发展的国内外差异—基于投融资并购及临床项目视角

董文政   

  1. 北京五加和分子医学研究所有限公司, 北京 100087
  • 收稿日期:2023-02-09 出版日期:2023-01-28 发布日期:2023-01-28

Differences in the Development of Cell and Gene Therapy Industry China and AbroadBased on the Perspective of Investment, Financing, M&A and Clinical Projects

  1. Beijing Five Plus Molecular Medicine Institute Co., Ltd., Beijing 100087, China
  • Received:2023-02-09 Online:2023-01-28 Published:2023-01-28

摘要: 目的:旨在探讨国内外细胞与基因治疗行业发展的特征、状态、模式及发展趋势方面的差异,探讨国内细胞与基因治疗行业发展过程中存在的不足以及未来需要着力解决的问题和相关对策。方法:按照融资(含并购、合作)-投资-临床项目的转化逻辑,选取2018—2022年各年度行业投融资并购纵向数据及目前临床项目的进展时点数据作为样本数据,进行细胞与基因治疗行业趋势分析和结构分析。结果和结论:国内外细胞与基因治疗行业发展在规模、增长方式及其结构、行业成熟度、市场逻辑、技术应用领域、治疗方式及其技术特点、临床适应证等方面差异显著。以美国为代表的国外细胞与基因治疗的商业成熟度已经达到较高水平,行业正处于快速成长期;其投融资并购集中体现为以先进基因治疗创新技术为驱动的商业逻辑,大量的罕见病基因治疗管线研发构成了细胞与基因治疗行业的金字塔底座;在临床项目中,以AAV为主的病毒载体体内基因治疗占据较高的比重。与之相较,国内细胞与基因治疗的商业成熟度较低,整体上处于成长的早期阶段,总体规模尚小,但发展速度很快;投融资并购集中体现为宽基市场应用和追求热点的市场导向逻辑,浮躁性、盲目性和跟随性比较突出;重产能、轻研发,上下游投融资配置不佳;在临床项目中,适应证分布不合理,针对恶性肿瘤的体外细胞基因治疗占绝对份额,且原创技术较低。但是,在层出不穷、大量的未被满足的临床需求面前,我国细胞与基因治疗后续将有巨大的发展空间,只要政策合理、引导得力,以先进基因治疗技术创新为特征的中国式细胞与基因治疗行业有可能再次加速中国制造业的崛起。
   

关键词: font-size:medium, ">细胞与基因治疗;行业发展;国内外;投融资并购;临床项目

Abstract: Objective:To explore the differences in the characteristics, status, mode and development trend of the development of the cell and gene therapy industry China and abroad, and to explore the deficiencies in the development process of the domestic cell and gene therapy industry, as well as the problems to be solved in the future and relevant countermeasures.Methods:According to the transformation logic of financing (including M&A and cooperation)-investment-clinical projects, the vertical data of industry investment and financing and the progress time point data of current clinical projects from 2018 to 2022 were selected as sample data for the trend analysis and structural analysis of the cell and gene therapy industry.Results and Conclusion:There are significant differences?in the development of cell and gene therapy industry China and abroad in terms of scale, growth mode and structure, industry maturity, market logic, technology application, treatment mode and its technical characteristics.The commercial maturity of cell and gene therapy abroad, represented by the United States, has reached a high level, and the industry is in a rapid growth period. Its investment and financing M&A are mainly reflected in the business logic driven by advanced gene therapy innovation technology. A large number of research and development of gene therapy pipelines for rare diseases constitute the pyramid base of the cell and gene therapy industry; In clinical projects, AAV-based viral vector gene therapy in vivo accounts for a high proportion.In contrast, the commercial maturity of cell and gene therapy in China is relatively low, and it is in the early stage of growth on the whole. The overall scale is still small, but the development speed is very fast.Investment and financing M&A are mainly reflected in the market-oriented logic of broad market application and pursuit of hot spots. The impetuosity, blindness and follow-through are relatively prominent, and the production capacity is more important than R&D, and the upstream and downstream investment and financing allocation is obviously deformed; In clinical projects, the distribution of indications is extremely unreasonable, the in vitro cell gene therapy for malignant tumors occupies an absolute share, and the original technology is low, with serious involution.However, in the face of an endless and large number of unmet clinical needs, China′s cell and gene therapy industry will have huge development space in the future. As long as the policy is reasonable and the guidance is effective, the Chinese-style cell and gene therapy industry characterized by advanced gene therapy technology innovation is likely to accelerate the rise of China′s manufacturing industry again.

Key words: font-size:medium, ">Cell and gene therapy; Industry development; China and abroad; Investment and financing M&, A; Clinical project

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