RNA疗法产业发展态势分析及建议 <sup>*</sup>

doi:10.13523/j.cb.2009037

中国生物工程杂志

2021, Vol. 41

Issue (2/3): 162-171 DOI: 10.13523/j.cb.2009037

行业分析

RNA疗法产业发展态势分析及建议 ^*

杨若南,许丽,徐萍,苏燕(

)

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

The Development Situation and Suggestions of RNA Therapy Industry

YANG Ruo-nan,XU Li,XU Ping,SU Yan(

)

Shanghai Information Center for Life Sciences, Shanghai Institute of Nutrition and Health,Chinese Academy of Sciences, Shanghai 200031, China

全文: PDF(15901 KB) HTML

摘要：

近年来,反义寡核苷酸药物、siRNA药物的相继获批上市,标志着 RNA疗法已逐渐从研究走向了产业化应用。目前RNA疗法已可应用于罕见病、肿瘤、感染性疾病等多种疾病的治疗。我国在RNA疗法产业上已有一定基础,但与国际相比,从事RNA疗法研发机构较少,研发管线单薄且缺乏核心技术,研发动力不足。对国内外RNA疗法领域产业发展态势进行梳理,分析我国RNA疗法的发展现状,提出发展对策建议。

关键词： ASO; siRNA; mRNA

Abstract:

In recent years, with a growing number of approved ASO and siRNA drugs, RNA therapy has gradually moved from clinical to industrial application, and RNA therapy currently can be applied to the treatment of rare diseases, tumors, infectious diseases and others. On the whole, a certain foundation on domestic RNA therapy industry has been built,while few engaged institutions, weak research pipelines and lacking of key technologies are still the main reasons that limit the development of RNA therapy industry in China. The development trends of RNA therapy industry at home and abroad are combed and analyzed, and then some suggestions for the development of RNA therapy in China are put forward.

Key words: ASO siRNA mRNA

收稿日期: 2020-09-27 出版日期: 2021-04-08

ZTFLH:

Q52

基金资助: * 国家重点研发计划(2016YFC0901904);上海市科委软科学项目资助项目(18692109100)

通讯作者: 苏燕 E-mail: suyan@sibs.ac.cn

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引用本文:

杨若南,许丽,徐萍,苏燕. RNA疗法产业发展态势分析及建议 ^*[J]. 中国生物工程杂志, 2021, 41(2/3): 162-171.

YANG Ruo-nan,XU Li,XU Ping,SU Yan. The Development Situation and Suggestions of RNA Therapy Industry. China Biotechnology, 2021, 41(2/3): 162-171.

链接本文:

https://manu60.magtech.com.cn/biotech/CN/10.13523/j.cb.2009037 或 https://manu60.magtech.com.cn/biotech/CN/Y2021/V41/I2/3/162

图1 RNA疗法技术体系图

图2 RNA疗法领域重要突破

图3 主要RNA疗法企业递送技术专利布局

图4 RNA疗法领域重要企业及相关合作企业

图5 目前已上市的各类RNA疗法药物

图6 RNA疗法在研药物数量

表1 已获批的ASO药物①

表2 ASO领域代表性公司临床Ⅲ期及NDA情况

表3 已获批的siRNA药物①

表4 siRNA疗法领域代表性公司临床Ⅲ期及NDA情况

表5 预防性mRNA疫苗领域代表性公司临床研究情况

表6 国内mRNA新冠疫苗研发进展

表8 美国Moderna公司全身性mRNA疗法临床研究情况

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