Research progress and CMC considerations on gene-editing medicinal products
LU Jia-qi
ZHOU Di
WEI Wei
HE Wu
Abstract：In recent years,gene-editing biotechnology products have shown great clinical therapeutic potential,but they are facing challenges such as insufficient accuracy,large editing efficiency fluctuations,and significant off target effects,which restrict their wide clinical application.At present,advanced technologies such as epigenetic editing and mitochondrial gene editing continue to develop,and the sequence and structure of gene editors are also rapidly iterating.The differences in the sequence design,structure,purity and other attributes of gene editors will affect the editing accuracy and precision and then the safety and effectiveness of the prducts.In the field of research and development and regulation,it is necessary to explore reasonable multidimensional quality assessment strategies and establish scientific and efficient drug regulatory tools.In this paper,the research progress and application status of gene-editing therapeutic drugs were investigated,and the consideration on gene editor design and quality evaluation was proposed based on the structures and purities of gene editing enzymes and sgRNA,efficiency and specificity of gene editing,so as to provide reference for the research and development and quality evaluation of such products.
Keywords：gene-editing medicinal productresearch progressCMC reviewquality study
Publication Date：2025-03-30
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：6( 608-613 )