On the afternoon of December 1^st, 2024, the Innovative Projects in Early Stage Roadshow of the 2024 China BioMed Innovation and Investment Conference (CBIIC) was successfully held at the Guangzhou Baiyun International Convention Center.

Yuan Qinghui, Head of Pharmaceutical and Biotechnology Industry Equity Research at China Securities.

The “Innovative Projects in Early Stage Roadshow” was first established at the 7th CBIIC as a important platform to showcase China’s original innovation capabilities and foster collaboration between innovative research and capital markets. This year, at the 9th CBIIC, the Innovative Projects in Early Stage Roadshow continued its previous momentum, focusing on early-stage drug projects in Phase I clinical trials or earlier, as well as cutting-edge research achievements in hot fields such as gene therapy, cell therapy, and novel drug delivery systems. After rigorous evaluation by the Roadshow Project Review Expert Committee, a total of 12 projects were presented at this year’s roadshow, covering therapeutic areas including neurodegenerative diseases, infectious diseases, oncology, insomnia, inflammatory bowel disease, and kidney diseases. Presenters shared insights from various perspectives, including technological highlights, market potential, team composition, and future development plans, effectively demonstrating the innovation and market competitiveness of their projects to investors, industry experts, and potential partners in attendance.

Project Highlights

Project 1: The Next-Gen Drug-Device Combination Product (Human Acellular Amniotic Membrane Scaffold + hUC-MSCs)

In recent years, China’s infertility rate has increased from 12% to 18%, with over 50 million couples currently affected by infertility. RGoodwill Cell Engineering Technology (Guangzhou) Co., Ltd. owns the only government-approved cell bank in Guangzhou, the Huadu Comprehensive Cell Bank, and is also the only cell therapy company in Guangzhou certified by the American AABB (for somatic cell processing, storage, and distribution of cell preparations). The company focuses on the development of cell therapy drugs in the fields of gynecology and reproductive health. CEO Zhang Xu of Goodwill Cell Engineering Technology introduced the company’s innovative self-developed “drug-device combination” procedure-human decellularized amniotic membrane scaffold combined with umbilical cord mesenchymal stem cells-highlighting the product’s technological advantages, efficacy, and development prospects. This aims to provide effective cell therapy for over 20 million patients with congenital and acquired uterine damage in China, thus improving the current infertility situation.

Presenter: Zhang Xu, CEO, Goodwill Cell Engineering Technology (Guangzhou) Co., Ltd.

Project 2: Transforming Duchenne Muscular Dystrophy Treatment by Delivering Next-Gen DMD Gene Therap

Dr. EJ Ma, Vice President of Business Development and External Innovation, CANbridge Pharmaceuticals, introduced the company’s gene therapy developed using dual adenovirus-associated vectors (Dual AAV). The therapy adopts the unique “StitchR” RNA assembly technology to achieve high fidelity and efficient trans-splicing, breaking through the capacity limitations of single AAV vectors and avoiding potential toxic side effects or assembly efficiency issues associated with other large gene assembly techniques. The therapy has been successfully validated in the DMD mouse model, showing superior efficacy, and is expected to become the world’s best gene therapy for DMD.

Presenter: EJ Ma, Vice President of Business Development and External Innovation, CANbridge Pharmaceuticals

Project 3: Innovative Broad-Spectrum Therapeutic Cancer Vaccine for Solid Tumors

Professor Huang Jiandong from the Department of Biomedical Sciences at the University of Hong Kong introduced BSTCV—the world’s first broad-spectrum and specific mRNA-based therapeutic tumor vaccine for solid tumors. The vaccine introduces microbial-derived antigens for the first time globally, transforming tumor cells into “foreign” cells, thus avoiding dependence on tumor neoantigen prediction. This innovative approach effectively avoids prediction biases and efficacy differences, rapidly activates existing immune memory, enhances tumor microenvironment remodeling, generates specific T-cells targeting multiple tumor mutations, and reduces the risk of immune evasion. Additionally, the vaccine has “on-demand” characteristics, is easy to mass-produce, cost-effective, and meets clinical demand. The project has completed preclinical concept verification and is preparing for pharmacological, toxicological, and pharmaceutical research stages.

Presenter: Huang Jiandong, Chair Professor of Synthetic Biology, Department of Biomedical Sciences, University of Hong Kong

Project 4: Development of a Highly Efficacious First-in-Class Vaginal Gel Targeting DRIPs for Cervical Cancer and Precancerous Lesions

Cervical cancer is the leading gynecological cancer, with 530,000 new cases and 250,000 deaths globally each year. There are no drugs for precancerous cervical lesions, and existing surgical treatments can lead to preterm birth, miscarriage, and have a high recurrence rate, presenting a huge unmet clinical need. Professor Liang Chun’s team at the Hong Kong University of Science and Technology discovered a new mechanism and pioneering target: inhibiting DRIPs, which can block cell proliferation from the source, induce apoptosis in precancerous lesions and cancer cells, while preserving normal cell survival. Ms. Zou Lan, co-founder and CEO of Encon Pharmaceuticals, shared the research team’s progress, noting that 17 patents have been granted for DRIPs inhibitors. The EN002 gel has already demonstrated its efficacy and safety in clinical trials for skin cancer and precancerous lesions. The proposed EN002 prodrug EK4-106 vaginal gel, with no systemic exposure, has completed preclinical efficacy and initial pharmacokinetics and safety evaluations, showing over 95% tumor suppression in vivo. The product is now in preclinical research.

Presenter: Ms. Zou Lan, Co-founder and CEO, Encon Pharmaceuticals

Project 5: NTQ5082—A Small-Molecule CFB Inhibitor with BIC Potential

Ji Xiaojun, Head of Translational Medicine at Jiangsu Chia Tai Tianqing Pharmaceutical Co., Ltd., shared the company’s development of NTQ5082, a small-molecule CFB inhibitor. The compound shows good CFB inhibition activity and high target selectivity. Preclinical studies have confirmed its ability to inhibit hemolysis in PNH patients’ red blood cells and significantly improve kidney function in animal models of IgA nephropathy and membranous nephropathy. Currently, NTQ5082 is undergoing Phase I clinical trials in healthy volunteers. Preliminary data indicates promising pharmacokinetics (PK)/pharmacodynamics (PD) characteristics and safety. Mr. Ji stated that NTQ5082 has BIC potential due to its activity, PK profile, and safety, and its broad indications make its market prospects highly promising. 

Presenter: Ji Xiaojun, Head of Translational Medicine, Jiangsu Chia Tai Tianqing Pharmaceutical Co., Ltd.

Project 6: A Novel Small Molecule Drug H021 Mediates miRNA, Potential to be the Best Treatment of IBD

H021, an oral small-molecule innovative drug independently developed by Jiangsu Carephar Pharmaceutical Co., Ltd., aims to address the current deficiencies in IBD therapies regarding efficacy, safety, and patient compliance. H021 upregulates miR-124 through its unique mechanism, regulating key immune factors such as TNF-α and IL-6, promoting immune balance, and showing great potential to become the best first-line therapy. Head-to-head preclinical studies showed that, compared to high-dose benchmark molecules, low-dose H021 was equally effective at inhibiting IL-6 levels and demonstrated differentiated advantages in reducing TNF-α levels, alleviating DAI scores, and improving colon pathology scores. Additionally, H021 showed lower impact on major metabolic liver enzymes and enriched in the gastrointestinal tract, indicating better safety and tolerance. Its Phase I clinical trial was initiated in Australia and China in the third quarter of 2024. The company plans to submit an IND to the U.S. FDA in 2025. With further development, H021 is expected to provide better treatment options for global IBD patients.

Presenter: Wang Chaolei, R&D Director, Jiangsu Carephar Pharmaceutical Co., Ltd.

Additionally, Mr. Huang Yue, CSO & COO of Plasmotact Therapeutics Limited HK, Professor Li Zhuorong from Institute of Medicinal Biotechnology, Chinese Academy of Medical Sciences, Dr. Liu Jianzhou from Peking Union Medical College Hospital, Associate Researcher Guo Qinglan from Institute of Materia Medica, Chinese Academy of Medical Sciences and Peking Union Medical College, Mr. Jason JI , CEO of Orisomes Biotech, and Mr. Howard FANG, Chief Development Officer of Shanghai Alpha Biopharma Co., Ltd., also presented their projects on-site, showcasing their innovative achievements and development potential to the attending investors and industry experts.

Huang Yue

Li Zhuorong 

Li Jianzhou

Guo Qinglan

Jason Ji

Howard Fang

The successful hosting of the Early Stage Project Roadshow at the 2024 CBIIC not only showcased the innovative vitality of the pharmaceutical field but also promoted the precise connection between innovative projects and capital, injecting a strong atmosphere of innovation into the entire conference. We look forward to seeing China’s pharmaceutical industry achieve even greater success in the future through continuous efforts and innovation.