Wu Xiaofei, Liu Shupeng, Liu Dan, Li Xiuqi, Wang Hongyun, Han Xiaohong
Clinical Pharmacology Research Center, Peking Union Medical College Hospital, State Key Laboratory of Complex Severe and Rare Diseases, NMPA Key Laboratory for Clinical Research and Evaluation of Drug, Beijing Key Laboratory of Clinical PK & PD Investigation for Innovative Drugs, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
Shenyang Pharmaceutical University, School of Life Science and Biopharmaceuticals, Shenyang 110000, China.
Pharmacol Res. 2024 Dec;210:107487. doi: 10.1016/j.phrs.2024.107487. Epub 2024 Oct 31.
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.
寡核苷酸药物有望引领制药创新的新浪潮,继小分子药物和单克隆抗体时代之后。本综述评估了该领域十年来在全球和中国的临床进展。自2013年以来,寡核苷酸药物的研发显著增加,尽管这些候选药物中的绝大多数仍处于临床试验的初期阶段。在全球范围内以及在中国,反义寡核苷酸(ASO)和小干扰RNA(siRNA)是两个关键类别。罕见病一直是寡核苷酸药物的主要治疗靶点,与全球趋势相比,中国在研项目中对其关注较少。同时,这些药物正在扩大其适用范围,涵盖多种适应症,可能会彻底改变慢性病的治疗方法。虽然与全球水平相比,中国在该领域的临床开发尚处于起步阶段,但技术进步和有利政策有望在未来推动寡核苷酸药物开发的新格局。