BioBAY Hosts Nucleic Acid Drug Development Forum, Charting the Course of the 'Third Wave' in Pharmaceuticals
On June 15, the Nucleic Acid Drug R&D Forum, hosted by BioBAY, was held in Shanghai. The forum focused on hot topics and investment trends related to nucleic acid drugs, including manufacturing processes, vectors, delivery technologies, and clinical development. It established a communication and exchange platform among government, industry, and research institutions to jointly explore the development and strategic layout of nucleic acid therapeutics, known as the “Third Wave of Pharmaceutical Innovation.”
The COVID-19 pandemic has brought nucleic acid technology into the spotlight. From nucleic acid testing, which shouldered the burden of nationwide screening, to nucleic acid vaccines, hailed as “the hope of the entire population,” these applications have demonstrated the pivotal role of nucleic acids in combating epidemic outbreaks. Unlike the mechanisms of traditional small-molecule chemical drugs and antibody-based therapeutics, nucleic acid drugs extend drug discovery to the genetic level, preceding protein targeting. By specifically upregulating or downregulating the expression of target genes, they address diseases that currently lack effective treatments, including genetic disorders and other refractory conditions. Consequently, nucleic acid therapeutics are regarded within the biopharmaceutical industry as the “third wave of drug development,” following small-molecule drugs and antibody-based therapies.
Yet challenges and opportunities coexist. After years of tortuous development, the critical bottleneck in nucleic acid therapeutics persists: key issues remain regarding how to ensure sufficient retention time for accurate targeting of pathological sites after injection into the human body, and how to avoid damage to normal cells. Furthermore, targeted delivery beyond the liver remains the greatest bottleneck. Ultimately, the development of efficient and safe drug delivery systems is essential for nucleic acid therapeutics to enter a new stage of advancement.
Against this backdrop, BioBAY initiated this forum. At the event,Ying Bobo, Founder and CEO of Suzhou Abogen BiosciencesA speaker shared insights on “Key Technologies and Clinical Development of mRNA Vaccines,” analyzing the advantages and challenges in mRNA vaccine R&D. Dr. Lu Yang, Chairman of Suzhou Sinotech Biologics, addressed key delivery technologies for nucleic acid drugs in a presentation titled “Progress of siRNA Drugs Delivered by Peptide Nanoparticles (PNP) in Tumor Treatment.” Yan Jinghua, a researcher at the Institute of Microbiology, Chinese Academy of Sciences, provided a detailed elaboration on “Antigen Design and Development of COVID-19 mRNA Vaccines.” Li Longcheng, Chairman of Zhongmei Ruikang, delivered a keynote speech on “Platform Technology Based on RNA Activation and Development of Small Nucleic Acid Drugs.” Zhang Peizhuo, Chairman and Chief Scientist of Genepharma, shared key priorities for the industrialization of nucleic acid drugs under the topic “Introduction to Process Development of Nucleic Acid Drugs.” Finally, Wang Jun, General Manager of the Investment Promotion Department at BioBAY, moderated a panel discussion with Ying Bo, Founder and CEO of Abogen Biosciences; Chen Jianxin, Chairman of Chuanxin Biotechnology; Li Longcheng, Chairman of Zhongmei Ruikang; Xie Ting, Executive Director at Hillhouse Capital; and Zou Jieyu, Managing Director at Lilly Asia Ventures. The panel engaged in in-depth exchanges and sharing on “Development Strategies for Nucleic Acid Drug Enterprises in China’s Capital Market.”
It is understood that while BioBAY continues to strengthen its foundation in antibody drugs, gene therapy, and cell therapy, nucleic acid therapeutics will emerge as a key strategic focus for future development. Currently, the park has gathered nearly twenty innovative enterprises in this field, forming an innovation ecosystem led by companies such as Abogen Biosciences, Sinotech Genomics, Advaccine Biotechnology, and GenePharma. Meanwhile, BioBAY is actively enhancing its industrial chain by introducing platform companies specializing in nucleic acid modification and delivery technologies, such as Trusina Biopharma, to accelerate the growth of innovative enterprises.
The event organizer, BioBAY, is a high-tech platform dedicated to incubating and developing the biopharmaceutical industry in Suzhou Industrial Park. After more than ten years of focused cultivation, the park has gathered over 500 innovative biopharmaceutical companies and nearly 15,000 high-level scientific and technological talents, forming three major industrial clusters: innovative drug R&D, high-end medical devices, and biotechnology. BioBAY is committed to building a world-class ecosystem for the biological industry.
Introduction to Leading Nucleic Acid Drug Companies at BioBAY:
Sirnaomics, a guest company in Village B, was among the first enterprises to settle in Village B in 2007. It is a biopharmaceutical company focused on the discovery and development of RNAi therapies for cancer and fibrotic diseases. The company advances its proprietary technology through the design of multivalent siRNA cocktails and nanoparticle-enhanced therapeutic delivery, establishing a deep presence in the treatment of tumors, fibrotic diseases, viral infections, and other conditions.
Currently, the product with the most advanced clinical progress in its pipeline is STP705, a small interfering RNA (siRNA) therapy. By leveraging its dual-targeting inhibitory properties and an optimized peptide nanoparticle (PNP) delivery system, STP705 directly targets the expression of two genes: TGF-β1 and COX-2. To date, STP705 has received FDA Orphan Drug Designation in the United States for three indications: primary sclerosing cholangitis, cholangiocarcinoma, and hepatocellular carcinoma (HCC), the most common type of primary liver cancer. In January this year, Sirnaomics announced the Phase 2a clinical trial results of STP705 for treating cutaneous squamous cell carcinoma in situ. The data showed that 76% of patients achieved histological complete clearance of tumor cells, while 90% of patients in the two optimal dose groups achieved histological complete clearance of tumor cells. These results not only successfully broke the curse of RNAi pharmaceutical companies failing in oncology drug development, but also led some clinicians to state that once approved, STP705 would be a “game changer.”
Abogen Biosciences, founded in Village B in early 2019, is an innovative biopharmaceutical company focused on the research and development of mRNA therapeutics. Since its establishment, the company has rapidly developed a proprietary delivery platform with independent intellectual property rights. This platform not only covers the field of infectious disease vaccines but also extends to tumor immunotherapy, treatment of rare diseases, and personalized cancer vaccines. Accordingly, in response to the sudden outbreak of the COVID-19 pandemic, the Abogen team swiftly initiated the development of a COVID-19 vaccine, leading to the creation of China’s first mRNA vaccine to enter clinical trials, thereby achieving a breakthrough from “0 to 1.”
In April this year, Abogen Biosciences announced the completion of its Series B financing round, raising RMB 600 million. The round was co-led by PICC Capital, SDIC Entrepreneurship Investment, and Yunfeng Capital, with participation from Hillhouse Venture Capital, Taifu Capital, Juming Venture Capital, Lifeline Capital, and Honghui Capital. The proceeds will be primarily used to accelerate the development of COVID-19 vaccines, establish a vaccine industrial base, and further expand the company’s product pipeline.
AiDiWeiXin, based in Village B, is a high-tech enterprise specializing in innovative vaccines. It possesses three internationally leading vaccine development platforms: the DNA vaccine platform, the recombinant protein subunit vaccine platform, and the adjuvant platform. Leveraging these technologies, the company has developed a pipeline of blockbuster candidate vaccines. Among them, the COVID-19 DNA vaccine, the novel respiratory syncytial virus (RSV) pneumonia vaccine, and the therapeutic hepatitis B vaccine have all demonstrated outstanding preclinical and clinical data.
Its core product is pGX9501 (INO-4800), a COVID-19 DNA vaccine jointly developed by Advaccine and the U.S.-based Inovio Pharmaceuticals. It is China’s first and only DNA-based COVID-19 candidate vaccine currently in clinical trials. The vaccine is expected to complete all clinical trials within this year and submit a marketing application to the Center for Drug Evaluation (CDE) of the National Medical Products Administration in the second half of the year.
Notably, in April this year, iVAC submitted its IPO prospectus to the Hong Kong Stock Exchange (HKEX), which was accepted, with China International Capital Corporation (CICC) serving as its exclusive sponsor. This makes iVAC the second biotechnology company to file for a listing in Hong Kong this year, following Transcenta Holding.
B-Cun Kejiguan Jima Gene, founded in B-Cun in 2007 by Dr. Zhang Peizhuo and his entrepreneurial team, is primarily engaged in the research and development, production, and application of biologics related to RNA research. The company also focuses on the development of RNA diagnostic reagents and innovative RNA-based therapeutics, establishing itself as one of the most comprehensive domestic suppliers of reagents and services for RNA research. Currently, the company operates a 14,000-square-meter facility dedicated to production and R&D, with a team of over 300 professionals. It has established integrated multi-platform capabilities encompassing RNA monomer and oligonucleotide synthesis, high-throughput screening of cells and tissues, metabolomics and genomics testing, as well as SPF animals, transgenic animals, and PDX models. This infrastructure enables the provision of comprehensive RNA research solutions. In addition to serving the domestic market, the company’s products are exported to overseas regions including Europe, the United States, and Japan.