Gene Editing Emerges as a Strategic Focus for Chinese Pharma Innovators, with Over 50 Companies Entering the Field

　　【Pharmaceutical Network Industry Dynamics] The field of gene editing is rapidly moving from laboratories to the forefront of industrialization, becoming a key focus for pharmaceutical innovation companies. In China, more than 50 companies, including RuiZheng Gene, YolTech Therapeutics, HuiDa Gene, Boya jiyin(Beijing)Biotechnology Co., Ltd., BRL Medicine, RuiFeng Bio, BenDao Gene, QiHan Bio, MeiJie Cell, DanRui China, BeiSi Bio, ZhengXu Bio, ShiXi Bio, ZhuanMa Bio, AiDiBeiKe, ZhongYin Technology, ShuTong Medical, YiJieLiKe, and XinYa Gene, have made strategic investments in this area, with continuous new advancements being reported.
 

On April 16, 2025, Rui Zheng Gene announced that its self-developed in vivo PCSK9 gene editing product ART002, which safely achieves saturated efficacy, completed the 24-week follow-up for all subjects with ultra-high baseline levels of low-density lipoprotein cholesterol (LDL-C) (>6mM) in an investigator-initiated trial (IIT) for the treatment of heterozygous familial hypercholesterolemia (HeFH). Clinical data showed that ART002 achieved saturated efficacy in knocking out the target protein PCSK9 in these subjects and maintained stable effects. Meanwhile, it safely and effectively reduced LDL-C levels, with an average reduction of 56% and a maximum individual reduction of up to 70%, which remained stable.
 

YOLT-101 Injection, an in vivo base editing drug targeting the PCSK9 gene developed by YolTech Therapeutics, received formal acceptance for its clinical trial application in April this year. The product utilizes the adenine base editor YolBE® and a novel lipid nanoparticle (LNP) delivery system. Unlike the DNA double-strand break mechanism relied upon by traditional CRISPR/Cas9 technology, YolBE® achieves precise single-base editing without causing DNA double-strand breaks. Preclinical studies have validated that this technology significantly reduces the risk of chromosomal abnormalities and off-target effects while demonstrating excellent genomic editing safety. This safety advantage enhances its potential application in treating common conditions such as cardiovascular diseases.
 

In addition, BRL-101 from BRL Medicine is a gene therapy product developed based on BRL Medicine's self-developed hematopoietic stem cell platform (ModiHSC). It is indicated for transfusion-dependent β-thalassemia and will soon enter Phase II clinical trials. RM-001 from Ruifeng Biotechnology is a self-developed autologous hematopoietic stem cell gene-editing product for the treatment of TDT. RM-001 uses CRISPR/Cas gene-editing technology to modify the γ-globin promoter, activating the synthesis of natural fetal hemoglobin in the human body, restoring red blood cells to their normal physiological function and eliminating the need for transfusions, thereby achieving the goal of treating β-thalassemia with a single dose. RM-001 is currently being studied in Phase I clinical trials. ET-01 from Boya Jiyin (Beijing) Biotechnology Co., Ltd., an autologous CD34+ hematopoietic stem cell injection with CRISPR/Cas9 gene-edited BCL11A erythroid enhancer, is an autologous, ex vivo gene-editing cell therapy for the treatment of TDT and is currently in Phase I clinical research.
 

The rapid development of China's gene editing industry is driven by continuous technological iteration and breakthroughs. The industry has witnessed three generations of updates in gene editing tools, including ZFNs, TALENs, and CRISPR technologies, significantly improving the efficiency, accuracy, and convenience of gene editing.
 

As enterprises continue to achieve clinical breakthroughs and sustain technological iteration, the industrialization prospects of China's gene-editing field are becoming increasingly clear. On one hand, the application of gene-editing technology in areas such as monogenic hereditary diseases, cancer, and cardiovascular diseases is deepening, offering the potential to solve medical challenges that traditional treatments struggle to address, indicating a vast market demand. On the other hand, Chinese companies have established a complete industrial chain layout from technology research and development, product development to clinical transformation. The "cluster effect" of over 50 enterprises not only enhances China's competitiveness in the global gene-editing arena but also provides substantial momentum for industry innovation.
 

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