Global and Chinese trends in oligonucleotide drug clinical development: A comparative analysis

IF 9.1 2区医学 Q1 PHARMACOLOGY & PHARMACY

Pharmacological research Pub Date : 2024-10-31 DOI:10.1016/j.phrs.2024.107487

Xiaofei Wu , Shupeng Liu , Dan Liu , Xiuqi Li , Hongyun Wang , Xiaohong Han

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Abstract

Oligonucleotide drugs are anticipated to mark the new wave of pharmaceutical innovation, succeeding the eras of small molecule drugs and monoclonal antibodies. This review assessed a decade of global and Chinese clinical advancements in this field. Since 2013, there has been a notable surge in the development of oligonucleotide drugs, although a considerable majority of these candidates are still in the nascent stages of clinical trials. Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) represent two pivotal classes both on global scale and within China. Rare diseases have been the main therapeutic target for oligonucleotide drugs, with a less pronounced focus in China's pipeline relative to the global trend. Concurrently, these drugs are broadening their scope to encompass a variety of indications, potentially revolutionizing treatment approaches for chronic conditions. While China's clinical development in this sector is in its infancy compared to the global stage, technological progress and favorable policies are expected to foster a new landscape of oligonucleotide drug development in the future.

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全球和中国的寡核苷酸药物临床开发趋势：比较分析。

继小分子药物和单克隆抗体时代之后，寡核苷酸药物有望成为医药创新的新浪潮。本综述评估了十年来全球和中国在这一领域取得的临床进展。自2013年以来，寡核苷酸药物的开发明显激增，尽管其中相当多的候选药物仍处于临床试验的初级阶段。反义寡核苷酸（ASOs）和小干扰RNAs（siRNAs）是全球和中国两大举足轻重的寡核苷酸类药物。罕见病一直是寡核苷酸类药物的主要治疗靶点，与全球趋势相比，中国的研发管线对这类药物的关注较少。与此同时，这类药物的适应症范围也在不断扩大，有可能彻底改变慢性病的治疗方法。虽然与全球相比，中国在这一领域的临床开发尚处于起步阶段，但技术进步和政策利好有望促进未来寡核苷酸药物开发的新格局。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pharmacological research 医学-药学

CiteScore

18.70

自引率

3.20%

发文量

491

审稿时长

8 days

期刊介绍： Pharmacological Research publishes cutting-edge articles in biomedical sciences to cover a broad range of topics that move the pharmacological field forward. Pharmacological research publishes articles on molecular, biochemical, translational, and clinical research (including clinical trials); it is proud of its rapid publication of accepted papers that comprises a dedicated, fast acceptance and publication track for high profile articles.