Early-Mover Oligonucleotide Therapeutics Paving New Paths for mRNA Innovation

With Alnylam’s total market capitalization surpassing $20 billion, the new wave of commercialization for small nucleic acid therapeutics has achieved remarkable success. In 2021, the company reported annual revenues of $662 million, with its flagship product ONPATTRO contributing $475 million. Alnylam’s strong favor among secondary market investors signals that small nucleic acid therapeutics have finally emerged as a significant and unignorable “major therapeutic area.”

In this niche sector, China has also reached a significant development milestone. Sirnaomics listed on the Hong Kong Stock Exchange, becoming the first Chinese RNA therapeutics company to go public. Nevertheless, in the field of small nucleic acid drugs, China’s industry is still catching up with global progress. This leaves several pressing questions for the domestic industry: What has been achieved by small nucleic acid drugs in recent years, fueled by the hype around mRNA? How can Chinese small nucleic acid drug developers mount a comeback amid core technology patent blockades? What is the cross-industry logic behind expanding small nucleic acid drug applications from liver diseases to obesity? Should companies build their own comprehensive platforms or pursue open collaborations—how to choose the R&D strategy that best fits their needs?

VCBeat New Medicine, in partnership with GTJA Investment, invites industry partners from the small nucleic acid drug sector to comprehensively and deeply share their insights on industry development through “report presentations” and “frontline discovery,” with the aim of accelerating the release of innovation.

Patrick Lu, Founder of Starnovo

He earned his Ph.D. from Sun Yat-sen University and subsequently conducted postdoctoral research at the University of Maryland and Georgetown University in the United States. He has received multiple R&D funding grants from the U.S. National Institutes of Health (NIH) and the Maryland State Government. In China, he led numerous scientific research fund projects, including major national special projects for new drug innovation during the 11th and 12th Five-Year Plan periods, and has been honored with several awards. With extensive practical experience in founding and managing multinational biopharmaceutical enterprises, he is a renowned expert in the field of international nucleic acid interference drug development and a serial entrepreneur. He founded Sirnaomics in 2007, which was listed on the Hong Kong Stock Exchange in 2021. He currently serves as Chairman of Sirnaomics, Inc. (USA) and RNAimmune, Inc. (USA) (Damian Bio).

Cui Wenhao, Chairman of Youjia Bio

Special Researcher at Osaka University, Ph.D. from Kyoto Prefectural University of Medicine, and Postdoctoral Fellow at Osaka City University. Studied under Professor Yasushi Kaneda at Osaka University and has been engaged in new drug development for 10 years. Has long maintained scientific research and clinical collaborations with universities and hospitals in Japan. Participated in the development of two innovative peptide drugs, as well as multiple gene-based therapeutics including small nucleic acid therapeutic vaccines and dendritic cell (DC) vaccines. His doctoral research achievements have completed Phase II clinical trials. He was awarded the Seiren Prize by Kyoto Prefectural University of Medicine in 2012 and is recognized as an expert under the Hangzhou Xiaoshan District “5213” Overseas Talent Program and the Hangzhou Major Overseas Talent Cooperation Project.

Zhao Xiaoyang, Head of Research Institute, Goteja Investment

Holds a Ph.D. from the Department of Surgery and Cancer, Faculty of Medicine, Imperial College London. An alumnus of Cambridge Judge Business School and CEIBS, and a member of the Shanghai Western Returned Scholars Association, the International Hepato-Pancreato-Biliary Association, the American Society for Gastrointestinal Endoscopy, the American Association for the Study of Liver Diseases, and the European Association for the Study of the Liver. Possesses profound and unique insights into the healthcare and pharmaceutical industries, has participated in and led multiple national-level entrepreneurship and innovation projects, maintains long-term observations of entrepreneurs in the healthcare sector, and has in-depth knowledge of the pharmaceutical equity investment market and industrial policies.

Sirnaomics is a clinical-stage biopharmaceutical company directly affiliated with the US-based Sirnaomics Group. Founded in 2008 by a team of returning overseas entrepreneurs led by Dr. Yang Lu, the company was listed on the Hong Kong Stock Exchange in 2021. The company focuses on building its nucleic acid interference drug delivery technology platform and researching and developing novel therapeutics. Leveraging its extensive experience in RNA interference (RNAi) technology, Sirnaomics has developed a unique nucleic acid interference drug delivery platform through its proprietary peptide nanoparticle (PNP) core technology. It has become an enterprise capable of simultaneously advancing clinical studies of novel RNAi drugs in both China and the United States, thereby promoting the progress of clinical trials for innovative RNAi therapeutics in China. The company has successively participated in major national-level projects for new drug innovation under China’s 11th, 12th, and 13th Five-Year Plans. It holds more than thirty authorized patents and patent applications in China, the United States, and Europe, establishing itself as a leading enterprise in the research and development of RNAi therapeutics in China.

Youjia Biopharma is a company dedicated to the research and development of novel oligonucleotide therapeutics. Co-founded by a team of overseas-returnee PhDs and professionals with extensive industrialization experience, the company is committed to its mission of “making medicines affordable for the general public and ensuring therapeutic options are available for diseases.” Its core focus lies in the research and development of innovative nucleic acid drugs with independent intellectual property rights. The company’s current pipeline covers fibrosis, antiviral therapies, cardiovascular diseases, and oncology. It has established a mature nucleic acid drug development platform, encompassing an intelligent design and screening system for nucleic acid therapeutics, delivery system development, and a CMC (Chemistry, Manufacturing, and Controls) platform.

Founded in 2001, GTJA Investment has been deeply engaged in the healthcare industry for over a decade. With a professional medical investment team, it has established a mature investment ecosystem platform for the healthcare sector. The firm has invested in more than 90 healthcare companies and successfully facilitated the public listing of 20 of them. Guided by in-depth industry insights, the GTJA Investment Research Institute launched its specialized industry research publication, *GTJA Insights*, in 2021 to share independent, objective value discoveries more accurately and timely. This issue of *GTJA Insights* (April 2022) provides an in-depth analysis of advancements in small nucleic acid drug technology, exploring the future of the industry together.

A media and research platform under VCBeat, focused on biopharmaceuticals, empowers innovators and entrepreneurs to disseminate value through both content and operations. In terms of content, it primarily features corporate profiles in frontier fields, investment and financing updates, R&D progress, reviews of outstanding domestic and international case studies, and industry reports on specialized sectors. On the operational front, through branded columns such as “VB Think Tank” and “VB Think Tank – Exclusive Report Launches,” it focuses on practical issues, discusses industrial trends, and fosters tangible collaborations through the exchange of ideas.

A flagship column under VBInsight. It features the inaugural release of in-depth research on high-growth sectors, accompanied by author commentaries, and invites industry practitioners as “Value Discovery Officers” to examine key challenges and solution frameworks from multiple perspectives. By combining report presentations with frontline insights, we contextualize practice through research and enrich research with fresh, real-world knowledge, jointly delivering innovative value with dedication.