Zhongyin Biotech Completes RMB 70 Million Pre-A Financing to Advance Ophthalmic Gene Therapy Pipeline into Clinical Development

VCBeat (WeChat ID: vcbeat) recently learned that Beijing CHIGENOVO Co., Ltd. (hereinafter referred to as “CHIGENOVO”) has completed a pre-A financing round of RMB 70 million,Led by Hetang Venture Capital, with follow-on investments from Longmen Capital and other institutional investors, the proceeds from the financing will be primarily used for GMP-grade virus production, pharmacology and toxicology studies, and clinical trials for the company’s ZVS101e product line, as well as to advance second-tier pipeline projects including ZVS203e, ZVS204e, ZVS105e, and ZVS106e. Previously, the company completed its angel round of financing in 2018.

CHIGENOVO is a national high-tech enterprise specializing in clinical genetic diagnosis, reproductive prevention, and the research and development of gene therapy drugs for hereditary eye diseases. It is also the first company in China to pioneer the R&D of ophthalmic gene therapy drugs. The company has established platforms for clinical genetic diagnosis of hereditary eye diseases, reproductive prevention coordination, and gene therapy drug development, dedicated to providing patients with hereditary eye diseases with comprehensive “one-stop” services encompassing precise genetic testing, reproductive prevention, and gene therapy.

Regarding securing this round of financing,Professor Yang Liping, Founder of CHIGENOVOIn response, they stated: “We appreciate the investors’ recognition of our team and their support for China’s efforts in blindness prevention and treatment. Hereditary retinal degeneration affects a large number of patients in China and is the leading cause of irreversible bilateral blindness. It represents a key focus of China’s blindness prevention and control initiatives, yet currently, there are no effective treatments available.”

Over the past two decades, the CHIGENOVO team has traveled across China to conduct surveys on hereditary retinal degeneration. Many families have fallen into poverty, and some have even been torn apart due to this disease. Therefore, the prevention and treatment of such conditions are not only scientific issues at the forefront of international academia but also significant social challenges facing the nation. However, drug development is a time-consuming and costly process, requiring substantial investments in human, material, and financial resources.

Inherited Retinal Dystrophies (IRDs) are a common group of monogenic blinding eye diseases, primarily including retinitis pigmentosa and Leber congenital amaurosis, among other types. They exhibit considerable heterogeneity in inheritance patterns, age of onset, and severity of visual impairment. Currently, therapeutic approaches for these conditions are mainly focused on gene therapy, which represents a key area of research interest in the field.

Gene therapy has achieved favorable outcomes in the treatment of inherited retinal degenerations (IRDs), a success directly attributable to the anatomical structure of the eye. First, the transparency of the ocular refractive media facilitates accurate injection of vectors under direct visualization. Second, the eye is anatomically relatively enclosed, ensuring that gene vectors transfect only specific target cells within the eye with minimal dissemination to other parts of the body. Third, the subretinal space exhibits a high degree of immune privilege, thereby largely preventing immune responses following injection. Consequently, gene therapy represents the sole current hope for restoring vision in patients with IRDs.

CHIGENOVO’s core team has been engaged in gene therapy research for many years, possessing extensive expertise and experience in basic research, clinical practice, AAV production, and drug regulatory submissions. In light of the significant heterogeneity in the pathogenesis of inherited retinal diseases (IRDs), CHIGENOVO has tailored its drug development strategies by focusing on common pathogenic genes among Chinese IRD patients. By integrating the specific pathogenic mechanisms of each gene, the company has separately designed gene replacement therapies and gene editing therapies.

The primary strategy of gene replacement therapy involves using recombinant adeno-associated virus (rAAV) vectors to deliver normal copies of mutated genes into patients’ retinal cells, thereby supplementing gene expression and achieving therapeutic effects. This approach is mainly targeted at genetic disorders caused by insufficient gene expression due to mutations. In contrast, gene editing therapy employs rAAV vectors to deliver repair sequences capable of correcting specific mutation sites into patients’ retinal cells, enabling precise correction of these sites and restoring normal gene function. This modality is primarily indicated for autosomal dominant genetic diseases.

CHIGENOVO's Clinical Pipeline

Leveraging its two core research platforms—gene replacement and gene editing therapies—CHIGENOVO has established four R&D pipelines for gene replacement therapy and two for gene editing therapy. Among these, the ZVS101e project is poised to officially initiate clinical trials, while ZVS203e and ZVS204e are currently being prepared for clinical trial applications.

Currently, CHIGENOVO has established more than 10 animal models of hereditary retinal degeneration, accumulated extensive experience in animal surgery and evaluation systems, and developed patient-derived iPSC reprogramming and 3D retinal organoid-based efficacy evaluation platforms. The company’s third-party clinical laboratory has amassed substantial genetic diagnostic data from patients with inherited retinal diseases (IRDs) and their family members, establishing a large-scale Chinese IRD cohort and database. By characterizing the spectrum of gene mutations in Chinese IRD patients, CHIGENOVO is developing gene therapies targeting pathogenic genes and mutation sites prevalent in this population, thereby addressing the specific needs of Chinese patients.

In addition to its gene therapy drug development platform, CHIGENOVO has also established a genetic diagnostic platform for hereditary eye diseases, providing patients with an integrated, closed-loop service encompassing pathogenic gene diagnosis, prevention, and gene therapy. Furthermore, the company’s independent drug development pipeline, designed based on its proprietary database, is well-positioned to meet the clinical diagnosis and treatment needs of the majority of patients.

“Gene therapy, as a cutting-edge therapeutic approach, initially targets monogenic genetic disorders and holds the potential for broader application across diverse disease areas in the future. With the advancement of new technologies, gene therapy will become a crucial option for treating various complex and refractory diseases. Overall, gene therapy offers immense potential,” summarized Professor Yang Liping. “CHIGENOVO is committed to providing affordable and highly effective medications for patients with hereditary eye diseases. Therefore, it is essential to remain focused and dedicated to excelling in this singular mission. In the field of gene therapy, we must delve deeply and thoroughly, ensuring professional strategic planning at every stage—from drug target discovery and validation of druggability to manufacturing platform support and clinical verification.”

Regarding technology-related aspects in investment,Lead Investor: Hetang Venture CapitalIt is evident that while the efficacy of gene therapy as a novel treatment modality has been clearly established, the technology still carries potential safety risks. Furthermore, given the current high cost of treatment, selecting appropriate therapeutic areas and indications is critical. Based on a comprehensive evaluation of clinical needs, technical barriers, safety, therapeutic efficacy, and production costs, we believe that ophthalmic genetic disorders represent one of the most promising directions for implementing gene therapy in China. Professor Yang Liping, founder of CHIGENOVO, has been engaged in the field of ophthalmic genetics for over two decades. With extensive clinical and research experience, she is a highly esteemed expert in this domain. The current selection of gene therapy targets is based on high-frequency loci identified from thousands of established family pedigrees, offering significant clinical application value and creating substantial technical barriers. In the current R&D pipeline, ZVS101e has demonstrated robust efficacy and safety in animal models and is poised to initiate exploratory clinical trials.

Longmen CapitalFounding Partner Wang HainingStatement: Gene therapy is one of the most cutting-edge frontiers in the life sciences sector and a key strategic focus for Longmen Capital. Over the past two years, we have invested in more than five gene therapy-related companies. CHIGENOVO possesses a comprehensive R&D platform for ophthalmic gene therapy and a robust pipeline of gene-based drug candidates, with its AAV vector platform ranking among the leading ones in China. Dedicated to treating monogenic hereditary eye diseases, CHIGENOVO has substantial technical reserves in both gene replacement therapy and gene editing therapy, with several products successively entering clinical trials. We look forward to witnessing swift clinical breakthroughs for CHIGENOVO’s products, enabling more patients to access domestically produced gene therapies.

Haitang Venture Capital Management (Beijing) Co., Ltd. (“Haitang VC”) was founded in 2001 and has long adhered to its mission of investing in technology-driven innovative enterprises. It focuses on high-tech, high-growth companies with core competitiveness during their start-up and growth stages, specializing in the incubation and investment of technology transfer and commercialization from Tsinghua University, covering fields such as information technology, life sciences, and clean technology. As an integral part of Tsinghua University’s innovation ecosystem, Haitang VC serves as the investment management platform for the university’s technology transfer and achievement commercialization initiatives. The Haitang series of funds currently managed by Haitang VC include the Haitang Exploration Fund, the Haitang Innovation Fund, and the Haitang International Health Fund, which focus respectively on the commercialization of scientific and technological achievements, regional industrial upgrading, and the healthcare industry. Throughout its development, Haitang VC has gained recognition from both domestic and international investors. Its fund investors include national and local provincial/municipal government guidance funds, financial institutions such as insurance companies and banks, state-owned enterprises, foreign enterprises, private enterprises, and high-net-worth individuals.

Longmen Capital, established in 2017, specializes in investments in innovative pharmaceuticals and life sciences, primarily targeting early-stage and growth-stage enterprises. The fund’s core team possesses extensive management and investment experience in the pharmaceutical sector. To date, it has launched three funds with a total assets under management (AUM) of RMB 2 billion. Longmen Capital has conducted rigorous screening and invested in over 30 high-quality companies, including Ailong Technology, Changfeng Pharmaceutical, Sirnaomics, Kyee Technology, Yusen Pharmaceutical, SwellBio, and Hode Biotherapeutics.