《生命科学》 2024, 36(1): 111-121
2023年基因治疗发展态势分析
摘 要：
基因治疗是指通过在基因水平上操纵或修饰细胞内基因的表达来治疗疾病的一种生物医学手段。近年来，基因治疗技术逐渐成熟，产业化加速，不断有重磅产品获批，已成为生物医药领域继小分子、大分子之后的一条新热门赛道。本文从基础研究、临床研究和产品获批等方面对2023 年基因治疗的态势进行了分析，结果发现治疗及递送新技术的不断涌现及疾病生物学研究的深入，推动基因治疗发展进入快车道，适应证范围不断拓展，临床潜力不断获得验证，产品加速上市。2023 年，基因替代治疗、基因编辑治疗和RNA 治疗等基因疗法先后迎来多款突破性新产品上市，递送技术的开发和优化取得重要进展，同时基因治疗领域潜在的安全风险和有效性仍需进一步的长期跟踪研究；基因治疗的可及性也有待多渠道来进一步提高。最后，本文也对基因治疗领域的未来发展进行了展望。
通讯作者：许 丽 , Email:xuli@sinh.ac.cn
Abstract:
Gene therapy refers to a biomedical means of treating diseases by manipulating or modifying the expression of cellular genes at the gene level. In recent years, with more products continuing to be approved, it has become a new hot track in the field of biomedicine after small molecules and macromolecules. This article analyses the development trend and progress of gene therapy in 2023 in terms of basic research, clinical research and product approval. The analysis found that the emergence of new technologies, as well as the deepening understanding of disease biology has pushed gene therapy into a new era, broadened the therapeutic scope of gene therapy, and accelerated the development of products. In 2023, a number of breakthrough new products of gene replacement therapy, gene editing therapy and RNA therapy have been launched. Significant progress has been achieved in the development and optimization of delivery technologies. Meanwhile, the potential risks and effectiveness of gene therapy still require further long-term tracking research. And accessibility of gene therapy also needs to be further improved by exploring multiple pathways. Finally, this paper also looks forward to the future development of the gene therapy.
Communication Author：XU Li , Email:xuli@sinh.ac.cn
