Genexen Gene Therapy Submits IPO Prospectus: A Leading Chinese CRO Specializing in Diverse Clinical-Grade Viral Vectors
In recent years, gene therapy has increasingly become a highly regarded innovative technology. With several gene therapy products successively approved in the United States and Europe, gene therapy has entered a phase of rapid development, emerging as the most promising direction in life sciences and healthcare.
According to statistics, as of August 2019, more than 3,100 gene therapy clinical trial protocols had been registered on the Clinical Trials website globally. Of these, over 1,400 had been declared completed, more than 400 were ongoing, and nearly 1,300 had been approved or were recruiting participants. Dr. Scott Gottlieb, former Commissioner of the U.S. Food and Drug Administration (FDA), stated at the 2018 BIO International Convention that the FDA was expected to approve 40 gene therapies by 2022.
In gene therapy, 70%–80% of therapeutic regimens are delivered via viral vectors. Viral vectors are key carriers for delivering therapeutic exogenous genes to target sites; they can be administered directly as injectable drugs or serve as critical raw materials in drug manufacturing.
As vectors for delivering exogenous genes, viral vectors must meet specific criteria: they should enable strict control over exogenous gene expression, achieve sustained and controllable therapeutic effects, exhibit target cell specificity, and have low toxicity and side effects. The manufacturing process for viral vectors is complex and costly, and quality control standards have not yet been standardized. This stands in stark contrast to the stringent requirements of gene therapy regarding viral vector quality (including viral titer and residual impurities) and dosage. Consequently, viral vectors are widely recognized as a major challenge and a key focus area for breakthroughs in in vivo gene therapy. The industrialization of viral vectors remains the most significant bottleneck preventing China’s gene therapy sector from catching up with that of the United States.
Currently, the majority of entities engaged in the research and development (R&D) of gene therapy drugs are research institutions and biotechnology companies, while the establishment and optimization of large-scale manufacturing processes required throughout the entire development-to-market journey are largely outsourced. In Shenzhen, Shenzhen Yuanxing Gene-tech Co., Ltd. has long provided contract research organization (CRO) services to gene therapy research institutions and enterprises, including preclinical pharmaceutical studies and clinical sample preparation for gene therapy products. Dr. Zhou Xiangjun, Chairman of Yuanxing Gene, told VCBeat that the company is leveraging its accumulated R&D and manufacturing expertise from over 60 viral vector-based gene therapy products to expand into full-industry-chain services, such as contract development and manufacturing organization (CDMO) and contract manufacturing organization (CMO) offerings.
Dr. Zhou Xiangjun is an expert in the field of gene therapy and studied in the United States for many years. In 2002, Dr. Zhou returned to China and joined Shenzhen Yuanxing Gene-tech Co., Ltd.
According to Dr. Zhou, Yuanxing Gene actually entered the field of gene therapy nearly 20 years ago and independently developed two gene therapy products. “At that time, alongside the R&D failures of some international peers, gene therapy was mired in considerable controversy, with many aspects failing to gain recognition.” Recalling the arduous explorations of gene therapy in clinical applications prior to 2010, Dr. Zhou still speaks with a sense of lament. Amidst an environment rife with doubts regarding safety and technical feasibility, the team ultimately shelved their R&D plans.
“Fortunately, by independently developing products in the early stages, the team gained a thorough understanding of the entire process of preclinical gene therapy product development, accumulated sufficient hardware capabilities, and established its own core technology platform.” Subsequently, research institutions and biotechnology companies in China successively sought to develop gene therapy products, creating an urgent demand for organizations that could provide outsourced services. In response, Shenzhen Yuanxing Gene-tech Co., Ltd. began to transform its business model to offer CRO services.
Shenzhen Yuanxing Gene-tech Co., Ltd.’s strategy is to closely follow the most advanced technologies and latest developments abroad, continuously exploring and optimizing processes through different products to achieve process standards consistent with those reported in cutting-edge literature.
The manufacturing process for gene therapy products, from viral vectors to final drug substances, involves several critical considerations, such as preventing viral vectors from regaining replicative competence, meeting yield and titer requirements, controlling residual impurities, and ensuring a sterile and pyrogen-free production process. Currently, although many companies are engaged in viral vector production, most applications are limited to scientific research, without comprehensive consideration and strict control of these factors. Therefore, there are currently few manufacturers capable of producing viral vectors that meet clinical requirements.
Yuanxing Gene focuses on manufacturing services for gene therapy-related products, primarily undertaking pharmaceutical research and clinical sample preparation for clients’ gene therapy products. Dr. Zhou Xiangjun told VCBeat that, in breaking through technical bottlenecks, Yuanxing Gene adopts a two-pronged approach: On one hand, it optimizes production processes, such as exploring serum-free suspension cell and virus culture techniques to address serum residue issues while enhancing viral titer and yield, thereby laying the groundwork for subsequent large-scale linear scale-up and commercial production. On the other hand, it continuously refines quality control methods to ensure product safety, purity, and potency. Furthermore, the company employs novel virus-packaging cell lines to reduce the probability of homologous recombination during viral production, thereby improving product safety.
The company’s technical platform is a versatile pharmaceutical technology platform dedicated to the development of manufacturing processes and quality standards for viral vector-based therapeutics. The platform demonstrates robust process stability and strong applicability; in addition to ensuring consistent product yield and quality, it enables the development of processes suitable for the production of other types of viral vectors.
To date, the majority of domestic enterprises and research institutions involved in gene therapy R&D have established business collaborations with Shenzhen Yuanxing Gene-tech Co., Ltd. to varying degrees. Among the CRO service projects provided by the company, the most advanced has entered Phase III clinical trials, while dozens of other candidates are at various stages of preclinical and clinical research. Additionally, some international clients have expressed interest in commissioning Shenzhen Yuanxing Gene-tech to handle the preparation of clinical samples for relevant viruses.
Dr. Zhou Xiangjun told VCBeat that, in addition to the rapid advancement in the CRO sector, Yuanxing Gene is also exploring the development of specific gene therapy products. This represents both an extension of the company’s business landscape from services to products and a return to the team’s original vision.
In terms of development strategy, Shenzhen Yuanxing Gene-tech Co., Ltd. will collaborate with foreign manufacturers to introduce corresponding products into China and secure the rights to these products in the Chinese market. The Special Administrative Measures for Foreign Investment Access (Negative List) issued by the National Development and Reform Commission of China prohibits foreign investment in the development and application of gene diagnosis and treatment technologies. This policy restricts the direct entry of foreign companies’ gene therapy products into China, thereby creating opportunities for collaboration with Shenzhen Yuanxing Gene-tech. Currently, Shenzhen Yuanxing Gene-tech has engaged in negotiations with several foreign companies regarding product introduction partnerships, although specific projects have not yet been disclosed.
It is understood that domestic investment firms such as Jinpu Capital, Tasly Capital, and Sinowood Capital have previously invested in Shenzhen Yuanxing Gene-tech Co., Ltd. The company is currently constructing a GMP-compliant production facility with a cleanroom area of nearly 10,000 square meters to expand its production capacity and meet the manufacturing demands for its marketed products. Meanwhile, Yuanxing Gene is planning to develop gene therapy products for rare diseases and is currently undertaking a new round of financing.