Objective: To provide suggestions and references for the pharmaceutical research of genome editing systems in advanced therapy medicinal products. Methods: Based on the pharmaceutical research, combined with the requirements of relevant regulation and guidance domestically and abroad, this article focused on the upstream design, production and quality research, use and risk management of genome editing systems. Results and Conclusion: In recent years, genome editing systems represented by CRISPR/Cas9 have risen rapidly as scientific research hotspot, and have been widely used in many fields such as medicine, agriculture, biotechnology and so on. In the field of therapeutics, genome editing systems can not only be used as active pharmaceutical ingredients or drug substances in vivo, but also play a therapeutic role after editing cells in vitro and returning to the human body. So it is an important concern in the evaluation of advanced therapy medicinal products. This article raised the current considerations for pharmaceutical research, hoping to promote the clinical transformation and application of genome editing products.
Yin Huifang
,
Wei Xuejing
,
Dai Yifei
,
Ma Xiaojuan
,
Zhang Jingchen
. Considerations for Pharmaceutical Research of Genome Editing Systems in Advanced Therapy Medicinal Products[J]. Chinese Pharmaceutical Affairs, 2025
, 39(5)
: 499
-505
.
DOI: 10.16153/j.1002-7777.2024-12-0021
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