HuiGene Therapeutics Completes Over RMB 100 Million Series A Financing Led by CDH Capital to Advance Gene Editing Therapies

VCBeat (WeChat ID: vcbeat) has learned that Hui-Gene Therapeutics (Shanghai) Co., Ltd. (hereinafter referred to as “Hui-Gene”), an emerging enterprise in gene editing and gene therapy, has announced the completion of its Series A financing round, raising over RMB 100 million. The round was led by Chende Capital, with participation from new investors WuXi AppTec, Huimei Capital, and Yahui Capital, as well as existing investor Sherpa Partners. The proceeds from this financing round will be primarily used to advance Hui-Gene’s core clinical pipeline, build R&D and preclinical research teams, and construct GMP-compliant production facilities.

Previously, Hui-Gene Therapeutics completed a RMB 30 million angel financing round from Sherpa Capital in November 2018.

Hui-Gene Therapeutics was registered and established in the Shanghai International Medical Zone on October 31, 2018, focusing on the research and development of novel therapeutic technologies and drugs for human rare genetic diseases. Adhering to gene editing technology innovation as its foundation and gene therapy R&D and production as its core, the company has now built a 500m²Gene Therapy R&D Laboratory (BSL-2), covering gene editing technology platform, AAV technology platform, disease model animal platform, and process translation and production platform, Phase II 5000m²²The gene therapy manufacturing facility is currently under planning and construction, with the aim of becoming the premier gene therapy platform in China and a world-leading one.

Dr. Yang Hui, the company’s founder, is a pioneer in the field of gene editing in China. He currently serves as a Principal Investigator at the Center for Excellence in Brain Science and Intelligence Technology of the Chinese Academy of Sciences and is a recipient of the National Science Fund for Distinguished Young Scholars. Dr. Yang has achieved a series of original breakthroughs in addressing the off-target safety concerns of novel gene-editing technologies, as well as in their applications in establishing animal models and treating diseases. In 2019 alone, he published significant findings on gene editing as the corresponding author in both Science and Nature. To actively promote the translation of basic scientific research into clinical applications and to apply novel gene-editing technologies to the gene therapy of genetic disorders, thereby benefiting more patients, Dr. Yang Hui co-founded Hui-Gene Therapeutics together with Dr. Yao Xuan and Dr. Shi Linyu.

Regarding this round of financing, Dr. Yao Xuan, Co-founder and CEO of Hui-Gene Therapeutics, stated that it was primarily attributable to the company’s two core strengths: its robust drug R&D translation capabilities and a fully closed-loop pipeline development process.

Dr. Xuan Yao, Co-founder and CEO of Hui-Gene

“Hui-Gene Therapeutics possesses robust R&D capabilities, enabling it to independently manage the entire process from research and development to commercialization,” explained Dr. Yao Xuan. “On one hand, Dr. Yang Hui, the founder of Hui-Gene Therapeutics, has dedicated years to optimizing gene editing systems and commands world-leading gene editing technologies. On the other hand, Dr. Shi Linyu, another co-founder, brings extensive expertise in constructing animal disease models and in the packaging and process translation of viral delivery vectors, particularly adeno-associated viruses (AAV). Furthermore, the majority of the company’s core team members have over a decade of experience in drug development.”

“On the other hand, leveraging our robust R&D capabilities, Hui-Gene Therapeutics is capable of independently executing the entire clinical development pipeline—from constructing animal disease models and developing gene-editing therapeutic strategies, to designing and engineering AAV delivery vectors, managing packaging and production, and conducting preclinical pharmacological studies in animals. We can achieve highly autonomous discovery and preclinical research of gene therapy drugs without relying on external CROs,” added Dr. Yao Xuan.

The development of the full-process clinical pipeline mentioned by Dr. Yao Xuan is underpinned by Hui-Gene Therapeutics’ four core technology platforms: the gene editing platform, the AAV technology platform, the disease animal model platform, and the process development and manufacturing platform.

Gene Editing Technology PlatformLeveraging the team’s strong background in gene editing technology and R&D innovation capabilities, we are able to perform CRISPR gene editing, DNA/RNA off-target detection, and DNA/RNA single-base editing (Base Editing), while promptly keeping pace with emerging gene editing tools;

AAV Technology PlatformThe team comprises researchers from world-class institutions such as MIT and Yale, with extensive expertise in AAV screening, engineering, packaging, and purification. The team is dedicated to developing AAV vectors characterized by high transduction efficiency, high tissue specificity, and low immunogenicity, thereby providing safe and effective delivery tools for gene therapy.

Disease Animal Model PlatformBy optimizing gene-editing technologies, we efficiently replace mouse genomic segments with human disease-causing gene mutations in situ, thereby rapidly and stably generating humanized mouse models that mimic the onset and progression of human diseases. This foundation enables the exploration and development of effective gene therapy strategies, providing critical therapeutic approaches and insights for subsequent drug development and treatment of related human genetic disorders. Furthermore, our team possesses extensive experience in preclinical animal studies and can independently conduct efficacy evaluation experiments, significantly accelerating the R&D process of gene therapy drugs.

Process Translation and Production PlatformSpecializing in GMP-grade viral vector packaging and production, the team has established technical barriers in both adherent and suspension cell systems, achieving a leading technological level in China. Phase I, with a capacity of 500 m², has been completed.²Gene Therapy Pilot-Scale Process Development Platform, featuring a team with extensive experience in biopharmaceutical process development and CMC, and equipped with a comprehensive quality management and regulatory compliance system. Phase II: 5,000 m²²The gene therapy manufacturing facility is currently under planning and construction, with production expected to commence in 2020.

As Dr. Yao Xuan noted, the core technical capabilities required for these technology platforms span a wide range, making it rare for a single enterprise to integrate them all. Even on the international stage, few companies possess all of these major technology platforms simultaneously, which constitutes the core competitiveness of Hui-Gene Therapeutics.

Supported by its four core technology platforms, Hui-Gene Therapeutics has rapidly advanced multiple key pipelines since its inception, spanning three major therapeutic areas: neurodegenerative diseases, visual impairments, and hearing impairments.

However, Dr. Yao Xuan also stated candidly that the company is not currently emphasizing the number of clinical pipeline programs initiated, but rather focusing on validating the entire R&D and manufacturing model for its pipelines to promote the maturation of its major technology platforms. Therefore, Hui-Gene Therapeutics has primarily selected monogenic rare genetic diseases as its initial clinical indications, aiming to directly repair defective genes using gene editing technologies, followed by process translation and production of gene editing therapeutics, with the ultimate goal of gradually expanding its indications from monogenic rare diseases to common diseases.

In the field of gene therapy, seven or eight drugs have already been approved by the FDA for market launch abroad, whereas in China, the industry remains largely at the stage of basic research. Addressing this gap between domestic and international development, Dr. Shi Linyu, Co-founder and CTO of Hui-Gene Therapeutics, shared his perspective: “In my view, the bottleneck hindering the development of gene therapy in China is the lack of GMP-grade AAV industrial production platforms. This is a critical challenge that must be addressed in the transition from scientific research to clinical application. In contrast, some overseas gene therapy companies already possessed mature industrialized platforms when they were acquired by large pharmaceutical enterprises.”

The importance of a GMP-grade AAV industrial production platform is self-evident. In the development of gene therapies, therapeutic agents rely on adeno-associated virus (AAV) vectors. As drug delivery vehicles, these viruses must first undergo safety testing and comply with Good Manufacturing Practice (GMP) standards. The required quantities of viral vectors vary depending on the specific disease being targeted. Therefore, achieving large-scale AAV production while ensuring safety represents a significant challenge in process development. It is reported that Hui-Gene Therapeutics is actively advancing the construction of its GMP-grade AAV industrial production platform. The involvement of Hui-Gene Therapeutics is expected to play a pivotal role in promoting the growth of China’s gene therapy market.

Lead Investor of This RoundZhu Tianji, Vice President of Chende CapitalExpressed strong confidence in the future of Hui-Gene Therapeutics: “We are excited about the groundbreaking work achieved by Professor Yang Hui and the Hui-Gene team in areas such as gene editing technology and vector screening. In this globally competitive field, the company’s team is undoubtedly at the forefront. Furthermore, in terms of clinical translation, the company possesses robust capabilities in editing and therapeutic strategies, vector design, and validation using humanized animal models, while its backend GMP-grade manufacturing processes have made significant progress over the past year. This combination of capabilities is extremely rare. Given this rapid growth trajectory, the company’s future is highly promising.”

Chende Capital is an investment firm focused on the life sciences and medical technology sectors, managing multiple USD and RMB funds. Led by a professional team with extensive experience in the healthcare industry, Chende Capital has completed investments in over 40 projects and achieved successful exits from several of them. The firm is committed to building top-tier companies in the healthcare sector and delivering optimal returns to its investors.

WuXi AppTec (Stock Codes: 603259.SH/2359.HK) provides comprehensive, integrated new drug research and development (R&D) and manufacturing services to the global biopharmaceutical industry. By empowering pharmaceutical, biotechnology, and medical device companies worldwide, WuXi AppTec is committed to accelerating the new drug R&D process and delivering breakthrough therapies to patients. Adhering to its mission of prioritizing research and maintaining a customer-centric approach, WuXi AppTec helps clients enhance R&D efficiency through cost-effective and high-efficiency services. Its service portfolio spans the R&D and manufacturing of small molecule drugs, cell and gene therapy R&D and manufacturing, and medical device testing.

Currently, WuXi AppTec’s enabling platforms are supporting R&D and innovation projects for more than 3,700 partners across over 30 countries worldwide. The company is committed to bringing more new and high-quality medicines to patients globally, striving to realize its vision of “making it easy to develop drugs and cure difficult diseases.”

HM Capital is a vertical fund within the Hillhouse Capital Group ecosystem, specializing in early-stage and growth-stage investments in healthcare. Leveraging the ecosystem advantages of Hillhouse Capital Group, Asia’s largest private equity firm, and the Mayo Clinic, a top-tier U.S. medical institution, HM Capital pursues multi-dimensional, holistic investments across the healthcare ecosystem. Committed to building a world-class industrial investment platform with a global perspective, HM Capital aims to help more early-stage and growth-stage healthcare companies achieve rapid expansion.

Yahui Investment is an early-stage investment fund focused on the life sciences sector. Centered on the application of cutting-edge innovative biotechnologies in disease diagnosis and treatment, Yahui Investment has completed investments in more than 20 early-stage biotech startups, with a portfolio spanning subsectors such as genetic testing, mass spectrometry testing, cell therapy, gene therapy, RNA therapeutics, and medical AI.

Sherpa Capital is a newly established specialized healthcare investment fund. Its core team members have collaborated for many years and possess extensive investment experience and an outstanding track record in China’s healthcare industry. The team is committed to serving as professional guides behind the climbers, empowering entrepreneurs to scale the peaks of their careers.