Zhongji Gene Therapy Secures RMB 110 Million Series A Financing to Advance Gene Therapy Drug Development with Over Two Decades of Expertise

VCBeat has learned that CNMDICR BIOPHARMA recently announced the completion of its RMB 110 million Series A financing round. The round was led by Longmen Capital, with continued participation from existing investor Enran Venture Capital, as well as co-investment from several other industry-renowned funds, including Puhua Capital and Haisong Medical Fund. Jianyihui (Chaoshan Capital) served as the exclusive financial advisor for this financing round. The proceeds will primarily be used to advance the Investigational New Drug (IND) application for the beta-thalassemia gene therapy project and to accelerate the research and development of gene therapies mediated by adeno-associated virus (AAV) vectors for recessive dystrophic epidermolysis bullosa (RDEB).

CNMDICR BIOPHARMA focuses on the field of classic gene therapy mediated by viral vectors and has laid out multiple gene therapy projects for monogenic genetic diseases. Its leading vector process, based on the concept of "virus-producing-virus," features high purity, high productivity, and high activity. Previously, CNMDICR BIOPHARMA completed a Pre-A financing round worth tens of millions of yuan, with investment from funds under Enran Venture Capital.

Gene therapy is one of the most promising frontier fields in the global pharmaceutical industry. According to statistics, there are currently seven gene therapy drugs approved for market launch worldwide, all of which are classic gene therapies mediated by viral vectors; globally, there are over 1,300 ongoing clinical trials for gene therapy. According to analysis by the renowned research firm Frost & Sullivan,The global gene therapy market is projected to reach USD 30.54 billion by 2025, with China’s market reaching RMB 17.89 billion.

Clinical research in gene therapy officially began in the 1990s. Although the field is currently experiencing robust growth, it has previously undergone significant fluctuations. The founding team of CNMDICR BIOPHARMA was personally involved when related research in China’s gene therapy sector was just getting underway, witnessing the entire journey from its inception and rapid expansion to a subsequent downturn, and finally to its revitalization.

The three founders of CNMDICR BIOPHARMA—Dong Wenji, Huang Yue, and Zhang Ye—enrolled in the doctoral program at Peking Union Medical College in 1997. They possess a well-defined academic lineage in gene therapy that traces back to the 1980s. After obtaining their PhDs, they pursued further studies at prestigious academic institutions in the United States or the United Kingdom. Upon returning to China, they all served as Principal Investigators at the Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences, where they conducted related research. Each founder has accumulated over two decades of continuous expertise in the fields of gene therapy and gene expression regulation.

Among them, Dr. Dong Wenji and Dr. Huang Yue studied under Academician Liu Depei in the same year,Dr. Wenji Dong specializes in gene therapy research for hemoglobinopathies and the development of viral vector technologies; Dr. Yue Huang focuses on the regulation of globin gene expression; and Dr. Ye Zhang has extensive experience in eukaryotic gene expression regulation and gene therapy.

Dr. Dong Wenji remarked, “At that time, the state had high hopes for gene therapy projects. The annual research funding for my team ranged from 250,000 to 350,000 yuan, and the outlook for gene therapy was very promising.” He further stated, “Unexpectedly, in 1999, the first patient death caused by gene therapy led to a sharp downturn in the global gene therapy field, with development nearly coming to a standstill.”

Affected by this event, the development of basic research and clinical applications in gene therapy worldwide rapidly declined. A large number of Chinese scientists in the field of gene therapy shifted their research directions, and Dr. Dong Wenji was no exception. However, he never abandoned his attention to and investment in gene therapy, continuing to advance research on viral vectors. “Even if viral vectors cannot become drugs themselves, they remain excellent application tools.”

With advancements in gene therapy technology and the concurrent promotion of supportive policies and regulations, particularly following the 2012 market launch of Glybera, the first gene therapy drug for a monogenic inherited disorder, basic research and clinical applications of gene therapy worldwide have entered a new phase of development. China’s gene therapy industry has also begun to revitalize, sparking a wave of gene therapy research.

Dr. Wenji Dong, Dr. Yue Huang, and Dr. Ye Zhang, three veterans in the field of gene therapy, co-founded CNMDICR BIOPHARMA based on their enduring passion for gene therapy and their profound professional expertise.Develop gene therapy drugs for severe monogenic genetic disorders that lack effective treatments.Currently, CNMDICR BIOPHARMA has established multiple R&D pipelines and has become a significant force in the field of gene therapy in China.

Dr. Dong Wenji told VCBeat that, based on his entrepreneurial experience and industry insights, the successful development of gene therapy drugs requires a team with comprehensive expertise in basic medicine and medical genetics, virology and vectorology, gene expression regulation, and the production of gene delivery systems.

1）In basic medicine and medical genetics,The application of gene therapy technologies is currently focused primarily on rare genetic disorders, requiring teams with extensive expertise in basic medicine and genetics. The core team at CNMDICR BIOPHARMA possesses both medical and genetic backgrounds, demonstrating a profound understanding of various genetic diseases.

2）In the field of virology and vectorology,Currently, the most commonly used viral vectors are AAV and lentivirus, with plasmid transfection typically employed as the production process. The team at CNMDICR BIOPHARMA has extensive expertise in virology and vectorology, possessing a thorough understanding of the biological backgrounds of various vectors, including baculovirus, adenovirus, lentivirus, and AAV. The company holds exclusive patents for its viral vector production systems.Production of Viral Vectors Using the Baculovirus-Insect Cell System, rather than using plasmid transfection, moreEfficient, Simple, Scalable. After the future product is launched, the company will have a significant advantage in industrialized production when competing commercially for high-volume drugs, such as those for muscular and neurological diseases that require direct systemic administration in vivo.

3）In contrast,Gene Expression RegulationThe importance of this factor is overlooked by most people; however, the CNMDICR BIOPHARMA team believes it may even be the most critical element in gene therapy. Only when genes are expressed at the right time, in the right spatial context, and at appropriate levels can the human body develop normally and acquire corresponding functions. Expression cassette design is a crucial component of gene therapy, as achieving appropriate gene expression in patients is key. Off-target expression in non-target cells, or excessively high or low expression levels, can all compromise the efficacy of gene therapy.

Dr. Huang Yue and Dr. Zhang Ye have conducted years of research on disease-related gene regulation. The CNMDICR BIOPHARMA team possesses significant advantages in technological expertise for gene expression regulation, enabling gene expression levels to more closely approximate normal states within a limited scope, thereby maximizing patient benefits. It is reported thatThe gene expression levels of candidate drugs in the company’s thalassemia pipeline approximate 70% of those observed under normal human physiological conditions.50% to 100% higher than existing published research and clinical data! In this scenario, the candidate drug clearly demonstrates superior efficacy and a broader patient eligibility profile.

4) Production and Preparation of Gene Delivery SystemsCMC (Chemistry, Manufacturing, and Controls), a concept derived from the traditional pharmaceutical industry that is frequently referenced within the sector, is of paramount importance. Currently, CNMDICR BIOPHARMA has successfully achieved AAV vector production at a 50-liter scale and lentiviral vector production at a 10-liter scale. Coupled with the pilot-scale manufacturing facility in Nanjing, which is nearing acceptance inspection, these capabilities fully meet the demands for clinical trials over the coming years. This proficiency positions the CNMDICR BIOPHARMA team’s CMC capabilities on par with leading domestic institutions.

Leveraging its comprehensive strengths in medical genetics, viral vectorology, gene expression regulation, and the production of gene delivery systems, CNMDICR BIOPHARMA has advanced steadily since its establishment, with an accelerating pace of development.The thalassemia pipeline is progressing smoothly, with ethical approval for the investigator-initiated trial (IIT) at the partner hospital already obtained. The research and development pipeline for recessive dystrophic epidermolysis bullosa (RDEB) has also received IIT ethical approval from the partner hospital. The company has three drugs poised to advance to the Investigational New Drug (IND) stage, and it is expected that 1–2 of these drugs will yield Phase II clinical trial data before 2024.

Dr. Dong Wenji summarized, “Currently, most domestic gene therapy companies are in a ‘follow-on’ position, with essentially all drugs in their pipelines already having comparable products launched abroad. Therefore, for Chinese enterprises to stand out, they must comprehensively and deeply invest in areas such as gene expression regulation, viral vectorology, and gene delivery systems to truly build core competitiveness and gain the capability to compete with leading overseas companies.”

“Furthermore, although the concept of the gene therapy market is generating significant enthusiasm, the industry as a whole remains in its early stages, with numerous challenges to be addressed across research and development, regulatory registration, and industrialization. In particular, more talent is needed to engage in relevant research on engineering viruses into vectors. It is hoped that enterprises, capital investors, universities, and laboratories will actively establish their presence in the field of gene therapy, thereby promoting its steady and sustained development.”

Mr. Wang Haining, Founding Partner of Longmen Capital“He stated, ‘Gene therapy is a hot sector within China’s biopharmaceutical industry and has been a key area of focus for me over the past two years. Broadly speaking, CAR-T therapies, nucleic acid drugs, and gene therapy products all fall under the umbrella of gene therapy. Within this broad domain, we have invested in nearly ten companies, each with differentiated approaches and solid foundational technologies. Even so, I am thrilled to have the opportunity to become an investor in CNMDICR BIOPHARMA.’”

“The founding team of CNMDICR BIOPHARMA consists entirely of professors from Peking Union Medical College, boasting years of accumulated expertise and heritage in basic research. Even more remarkably, they have been dedicated to the industrialization of gene therapy. Their innovative viral vector manufacturing process, based on the ‘virus-producing-virus’ concept, can significantly reduce the cost of viral vectors, enhance the accessibility of gene therapy, and benefit a larger patient population.”

Ms. Zhu Baolin, Vice President of Investment at Puhua Capital“Gene therapy holds the promise of a cure for patients, and has long been a key focus area for Puhua Capital. We have already invested in multiple CGT projects, and we are delighted to participate in this round of financing for CNMDICR BIOPHARMA.”

“Although the commercialization of CGT is still in its early stages, there are already thousands of R&D projects underway, and CNMDICR BIOPHARMA has established its own differentiated advantages. We are optimistic that the team’s profound expertise in basic medicine and medical genetics will provide a solid scientific foundation for the development of innovative products. Furthermore, from a commercial perspective, the company’s patented virus production concept offers a potential solution to the persistently high pricing of gene therapies, which is expected to further improve accessibility and bring hope to more patients. We look forward to the steady progress of the company’s R&D efforts and the early market launch of its products to create value for society.”

Mr. Sun Jigang, Founding Partner of Haisong Medical Fund“Haisong Healthcare Fund is delighted to have the opportunity to co-invest in this round of financing for CNMDICR BIOPHARMA alongside several other outstanding funds in the industry. Cell and gene therapy has long been a key focus area for Haisong Healthcare Fund, which has already invested in numerous high-quality companies in this sector. Thalassemia represents a clear unmet clinical need with a large patient population, creating favorable conditions for the future commercialization of gene therapies. The CNMDICR BIOPHARMA team has accumulated decades of experience and investment in foundational clinical research, offering unique insights into gene drug development—a testament to their profound expertise and steady growth. We look forward to the early market launch of CNMDICR BIOPHARMA’s 100% independently developed gene therapy, bringing hope to the many patients with rare diseases in China.”

Finally, regarding this investment,Dr. Chen Renhai, Founding and Managing Partner of Enran Venture Capital“Following our exclusive angel investment, we are delighted to continue supporting CNMDICR BIOPHARMA at this critical stage of its development. The company boasts an outstanding core team, leading-edge core technologies, sustained innovation capabilities, and remarkable execution in advancing its pipeline. In particular, its profound expertise in gene expression regulation and unique viral vector manufacturing processes, combined with highly valuable innovative achievements, provide stable and enduring momentum for the company’s long-term growth. Enran Venture Capital has always been committed to becoming a professional investment institution in the biopharmaceutical and healthcare sectors, focusing on the commercial value generated by the integration of ‘life,’ ‘health,’ and ‘innovation.’ Moving forward, Enran will fully leverage its resources in the biopharmaceutical field to help CNMDICR BIOPHARMA become a world-class gene therapy company.”

Longmen Capital is a specialized venture capital firm in the pharmaceutical industry, focusing on early-stage and growth-stage investments in innovative pharmaceuticals and biotechnology. The team members all have medical or biological backgrounds and possess many years of experience in pharmaceutical investment and industry operations. Since its establishment in 2017, Longmen Capital has invested in more than 30 pharmaceutical companies, including Changfeng Pharmaceutical, Stmicro (Swei Microbiology), Sirnaomics, Kyee Technology, Huade Biologics, and CNMDICR BIOPHARMA, with assets under management reaching RMB 2 billion. Longmen Capital has always adhered to the principles of independent research and value discovery, serving as an early investor for many innovative enterprises, aiming to promote the industrialization of scientific and technological achievements through capital.

Puhua Capital, founded in 2004 and headquartered in Hangzhou, is a professional venture capital and asset management firm with investment offices in Beijing, Shanghai, Shenzhen, Xiamen, Chengdu, and London, UK. Since its inception, Puhua Capital has adhered to its vision of “Upholding Universal Benevolence, Pursuing Substantive Excellence,” investing in early-stage and growth-stage innovative startups to help entrepreneurs realize their dreams while striving to generate robust returns for investors. Leveraging its profound industry insights and extensive capital market experience, Puhua has established a dual-drive investment strategy of “Capital + Industry,” rooting itself in industrial sectors and deepening regional engagement. It focuses on venture investments in new technologies, healthcare, culture and consumption, and new energy. Over the past 17 years, Puhua Capital has supported more than 400 startups, with over 70 achieving exits through IPOs, mergers and acquisitions, and other channels.

Haishong Healthcare Fund was established in 2016, with cumulative assets under management exceeding RMB 5 billion. It currently maintains offices in Beijing, Nanjing, and Suzhou. As a specialized venture capital fund under Haishong Capital, Haishong Healthcare Fund focuses on identifying innovative companies both domestically and internationally that possess unique advanced technologies, independent R&D capabilities, and strong market growth potential. The fund prioritizes investment opportunities in cutting-edge healthcare sectors, including biopharmaceuticals, advanced cell and gene therapies, innovative medical devices, regenerative medicine technologies, and AI-driven healthcare solutions. Its portfolio includes high-quality domestic and international projects such as Edigene (gene editing), Huadao Biotherapy (CAR-T), iRay Technology (flat-panel detectors, listed on the STAR Market), Hui Naoyun (AI), Weilitop Biotech (antibody drug development), Accutar Biotech (small-molecule anti-tumor drugs), and Zhimeng Biopharma (anti-hepatitis B virus).

Enran Venture Capital was established in 2015. It strategically invests in revolutionary new technologies and products across the global biomedical and healthcare industry chains, striving to become a specialized investment institution in the biomedical and healthcare sectors. The company’s management team and investors possess extensive investment experience and deep industrial resources in the biomedical and healthcare fields, while focusing on the commercial value generated by the integration of “life,” “health,” and “innovation.” Enran Venture Capital specializes in life sciences, healthcare, and innovation, and is committed to serving as an investor, partner, and co-founder to enterprises.