Why is nucleic acid testing so popular? The underlying logic of capital betting
In 2026, the biopharmaceutical industry will enter a critical stage of innovation iteration, and the small nucleic acid track will become the core focus of industry attention with technological breakthroughs, clinical achievements landing, and continuous capital support. From the niche field of rare disease treatment to the vast market of chronic disease treatment, small nucleic acid drugs have gradually expanded from technology catch-up to achieving global integration and even leading in some fields. From early research and development financing to cross-border enterprise mergers and acquisitions, small nucleic acid drugs are reshaping the global pharmaceutical innovation pattern with the posture of the “third-generation pharmaceutical revolution”.
This article will systematically analyze the underlying logic of the outbreak in the small nucleic acid industry, comprehensively review the financing and trading situation in the global and Chinese small nucleic acid fields in 2026, and deeply analyze the development trends and potential opportunities of the industry.
1ć The underlying logic behind the outbreak of the small nucleic acid industry: from technological breakthroughs to industrial maturity
ļ¼1ļ¼ The core definition and disruptive advantages of small nucleic acid drugs
Small Nucleic Acid Drugs are a class of short chain nucleic acid molecules consisting of 10-30 nucleotides, mainly including Small Interference RNA (siRNA), Anti Sense Oligonucleotides (ASO), MicroRNAs (miRNA), etc. Their core mechanism of action is to precisely regulate the expression of pathogenic genes at the mRNA transcription level, blocking the disease progression from the source. Compared with traditional drugs, they have significant disruptive advantages.
Compared with the first generation of small molecule drugs (targeting proteins, easily developing drug resistance, about 80% of targets cannot be used as drugs) and the second generation of antibody drugs (large molecular structure, difficult to achieve intracellular targeting, high frequency of administration), the core advantages of small nucleic acid drugs are reflected in three aspects:
1. Comprehensive target coverage, breaking through the bottleneck of “non drug”: theoretically, it can target all human genes, providing new treatment pathways for diseases that traditional drugs cannot cover, greatly expanding the boundaries of drug development;
2. High targeting accuracy and controllable off target risk: relying on the principle of complementary base pairing, it can specifically identify and bind to target genes. Compared with traditional drugs, its off target risk is significantly reduced, and clinical side effects are easier to control;
3. Outstanding long-term effectiveness and improved patient medication compliance: After chemical modification and delivery technology optimization, the efficacy of small nucleic acid drugs can be maintained for 3-12 months. The traditional “daily/weekly administration” mode for chronic disease treatment has been optimized to “one dose every six months/year”, effectively solving the industry pain point of insufficient medication compliance among chronic disease patients.
ļ¼2ļ¼ The triple core driving force behind the outbreak of the small nucleic acid industry in 2026
1. Breakthrough in delivery and modification technology
The core bottleneck of the industrialization of small nucleic acid drugs lies in the delivery system, that is, how to accurately deliver fragile nucleic acid molecules to target tissues, avoid in vivo degradation, and achieve efficient cellular entry. In 2026, several key breakthroughs were made in the delivery and chemical modification technology of small nuclear acids, completely breaking the technological constraints of industrial development
GalNAc liver targeting technology is becoming increasingly mature: the coupling technology of N-acetylglucosamine (GalNAc) and siRNA can accurately target liver cell ASGPR receptors. Currently, six siRNA drugs have been successfully launched, becoming the “cornerstone technology” for the commercialization of small nucleic acid drugs;
Global breakthrough in extrahepatic delivery technology: Chinese companies have shown outstanding performance in this field, among which the Shengyin Biotechnology LEAD platform can achieve targeted delivery of multiple tissues such as fat, muscle, and nerve, the Hejiya Biotechnology MVIP platform has achieved the technical goal of “one dose per year” for long-term delivery in the liver, and the Da Rui Biotechnology GAIA ā¢ The platform effectively reduces off target risks and further enhances drug safety;
Continuous innovation in chemical modification and dual target technology: The application of chemical modification techniques such as 2 ‘- O-methyl and phosphothio skeleton has significantly improved the in vivo stability of small nucleic acid molecules; The dual target siRNA technology can synchronously regulate two pathogenic genes, effectively improving drug efficacy and expanding the coverage of indications.
2. Expansion of indications and continuous realization of commercial value
Early small nucleic acid drugs mainly focused on rare diseases such as SMA and ATTR, with relatively limited market space. In 2026, the clinical breakthrough of small nucleic acid drugs in the field of large indications will become the core catalyst driving the industry outbreak:
In the field of cardiovascular metabolism:
As the largest market for the application of small nucleic acid drugs, Novartis’ Envision (siRNA based lipid-lowering drug) sales will exceed 750 million US dollars in 2025, a year-on-year increase of 112%; The siRNA based antihypertensive drug developed by Alnylam in collaboration with Zilebesiran has successfully completed Phase 3 clinical trials, achieving a long-lasting pressure control effect of “one injection every three months”, and is expected to tap into the billion dollar hypertension treatment market;
In the field of weight loss metabolism:
Two siRNA weight loss drugs developed by Arrowhead have shown excellent clinical data, with significant weight loss effects as single drugs. When used in combination with GLP-1 drugs, the weight loss effect can be doubled, while effectively avoiding the clinical pain point of muscle loss;
In the field of kidney disease and liver disease:
RG002C0106 (siRNA based drug) developed by Xuanjing Biotechnology has entered phase 2 clinical trials and is expected to become the world’s first small nucleic acid drug for IgA nephropathy; RBD1016 targets hepatitis B virus, providing a new possibility for functional cure of hepatitis B;
In the field of neurology and rare diseases:
ASO drugs have been continuously validated for clinical value in the treatment of diseases such as SMA and Huntington’s disease, with significantly higher clinical conversion rates than traditional drugs, providing more effective treatment options for rare disease patients.
3. Policy and Market: Global Synergy, China Becomes Core Growth Pole
The United States and the European Union have listed small nucleic acid technology as a strategic direction for pharmaceutical innovation, and have promoted the commercialization of small nucleic acid drugs through policies such as accelerating review and approval, expanding medical insurance coverage, etc; The 14th Five Year Plan for Bioeconomy has listed nucleic acid drugs as a key development area, and many regions have introduced special policies to support the research and development of core technologies such as extrahepatic delivery. The clinical evaluation cycle for small nucleic acid drugs has been reduced by more than 50% compared to before, significantly improving the efficiency of research and development and market launch;
The global small nucleic acid drug market has entered an explosive period. The global small nucleic acid drug market has shown strong and sustained growth, increasing from $2.7 billion in 2019 to $5.7 billion in 2024, with a compound annual growth rate of 16.2%. Driven by continuous technological advancements, increasing market approvals, and clinical validations, it is expected that the global small nucleic acid drug market will accelerate its growth, reaching $20.6 billion and $54.9 billion in 2029 and 2034, respectively. The compound annual growth rate from 2024 to 2029 is 29.4%, and the compound annual growth rate from 2029 to 2034 is 21.6%. China has become the world’s second-largest nucleic acid market after the United States, with the number of domestically produced small nucleic acid drug pipelines ranking first in the world.
2ć The underlying logic of financing in the small nucleic acid field in 2026
ļ¼1ļ¼ Global Financing Landscape: Multinational Pharmaceutical Companies Accelerate Layout, Transaction Scale Reaches Historical High
In 2026, the global small nucleic acid field will enter a dual drive stage of “giant integration+innovative enterprise explosion”. Multinational pharmaceutical companies (MNCs) will accelerate their layout through acquisitions, authorization cooperation, and other means, and the industry’s transaction scale and frequency will reach a historical high
Novartis, as a leading enterprise in the field of small nucleic acid, has improved its entire pipeline layout through a series of mergers and acquisitions. It has acquired Leqvio for $9.7 billion, Regulus for $1.7 billion, and DTxPharma for $1 billion, building a full range of small nucleic acid drug matrices covering RNAi, ASO, and miRNA;
Roche/Genentech reached a cooperation agreement with Sinovac Biotech in February 2026, obtaining authorization for Sinovac Biotech’s extrahepatic delivery heavy-duty pipeline through a $200 million down payment and a $1.5 billion milestone, demonstrating the international giant’s recognition of China’s small nucleic acid technology;
Multinational pharmaceutical companies such as Eli Lilly, Merck, and GSK have intensively laid out small nucleic acid enterprises in China. By early 2026, the potential total value of three BD transactions for Chinese small nucleic acid enterprises exceeded 7 billion US dollars, breaking the record for domestic innovative drug overseas transactions;
Industry data shows that from 2025 to 2026, there will be over 40 BD transactions in the global small nucleic acid field, with a potential total transaction value of over 70 billion US dollars, an increase of over 8 times compared to 2023, reflecting the high attention of global capital to the small nucleic acid track.
ļ¼2ļ¼ China’s financing inventory: IPO+M&A+private equity all erupt across the board
1. IPO milestone: Ruibo Biotechnology has landed on the Hong Kong Stock Exchange, leading the capitalization process of domestic small nucleic acid enterprises
On January 9th, Ruibo Biotechnology (06938. HK) officially landed on the Hong Kong Stock Exchange, becoming an important benchmark for capitalization in China’s small nucleic acid field and setting three industry records:
As the first innovative drug IPO in the Hong Kong stock market in 2026, Ruibo Biotechnology issued at a price of HKD 57.97, raising a total of over HKD 1.8 billion;
On the first day of listing, the stock price rose by 41.62%, the market value exceeded HKD 13.6 billion, and the public offering subscription multiple exceeded 100 times, reflecting the high recognition of the capital market for the small nucleic acid track;
As a leading enterprise in the field of small nucleic acids in China, the core pipeline of RiboGalSTARTM covers such core fields as lipid-lowering, hepatitis B and MASH. Its RiboGalSTARTM platform technology has reached the world’s leading level. In 2023, it reached a cooperation agreement of 2 billion dollars with Boehringer Ingelheim, demonstrating its technical strength and commercial potential.
2. Private equity financing: Normalization of billion yuan level financing, steady increase in early project valuations
From January to March 2026, financing in the field of small nucleic acid testing in China showed the characteristics of “large quantity, large amount, and wide coverage”. There were 12 financing cases exceeding 100 million yuan, with a total financing amount exceeding 5 billion yuan. The valuation of early projects (Pre-A/A round) generally exceeded 1 billion yuan. The specific financing situation is shown in the table below:
3. Heavy BD transactions: Domestic technology has gained international recognition, and the scale of overseas transactions continues to break through
At the beginning of 2026, Chinese small nucleic acid enterprises reached multiple heavyweight BD transactions with multinational pharmaceutical companies, with transaction volumes continuously breaking records, marking the recognition of domestic small nucleic acid technology in the international top market. The specific transaction situation is as follows:
1. Shengyin Biotechnology and Genentech: The two parties have reached a pipeline authorization cooperation. Genentech has obtained the global authorization for Shengyin Biotechnology’s extrahepatic delivery of siRNA pipeline through a $200 million down payment and a $1.5 billion milestone, with a total transaction amount of $1.7 billion;
2. Ruibo Biotechnology and Madrigal: Reached authorization cooperation around the dual target siRNA pipeline in the MASH field, with a transaction amount of $60 million down payment+$3.44 billion milestone, and a total amount of $4.4 billion, breaking the record for domestic small nucleic acid transactions;
3. Frontier Biotech and GSK: The two parties have reached an authorized cooperation on two small nucleic acid pipelines, with a transaction amount of $40 million down payment and a milestone of $947 million, totaling nearly $1 billion, further promoting domestic small nucleic acid technology to go global.
3ć Analysis of the Core Competitiveness of the Small Nucleic Acid Race: Highlighting the Advantages Compared to CGT and PROTAC
ļ¼1ļ¼ Comparison with CGT (Cell and Gene Therapy): Commercialization maturity has more advantages
Although CGT technology is cutting-edge, it has problems such as high clinical risks, complex production processes, expensive treatment prices, and narrow indication coverage. Currently, only a few CGT products worldwide have achieved profitability;
Small nucleic acid drugs have advantages such as high clinical success rates (with a conversion rate of over 60% from phase I to III, significantly higher than the 30% of small molecule drugs), standardized production processes, controllable treatment prices, and full coverage of indications from rare diseases to chronic diseases. In 2026, multiple small nucleic acid drugs will enter the commercial revenue period, with strong cash flow certainty and better risk return ratio.
ļ¼2ļ¼ Comparison with PROTAC (Protein Degradation Targeted Chimera): Significant advantages in target space and long-term efficacy
PROTAC technology mainly focuses on protein degradation and is still limited to “druggable targets”, with problems such as high frequency of administration, high risk of off target, and difficulty in oral administration;
Small nucleic acid drugs can target all human genes and have the advantages of long-term sustained release and subcutaneous injection, forming a “dimensionality reduction blow” in the field of chronic disease treatment. Compared with the daily oral administration of traditional drugs, the “six month one shot” mode of small nucleic acid drugs significantly improves patient medication compliance and expands market space.
4ć Outlook on the Development Trends of the Small Nucleic Acid Industry from 2026 to 2030
ļ¼1ļ¼ Market size continues to grow: synchronous expansion of global and Chinese markets
According to experts’ predictions, the global market size of small nucleic acid drugs will reach 54.9 billion US dollars by 2030, with a compound annual growth rate (CAGR) of 21.6% from 2026 to 2030; The size of China’s small nucleic acid market is expected to exceed 16 billion US dollars, accounting for over 30% of the global market and becoming the largest single small nucleic acid market in the world, with enormous growth potential for the industry.
ļ¼2ļ¼ Next generation small nucleic acid technology leads industry development
In the next five years, small nucleic acid technology will continue to iterate and upgrade towards higher targeting, higher delivery efficiency, and wider coverage of indications, presenting three main development lines of precision, multifunctionality, and intelligence. Specifically, it will focus on the following key areas:
Delivery technology 2.0 fully upgraded, breaking through the bottleneck of targeting extrahepatic tissues
The liver targeting technology represented by GalNAc has achieved mature applications, but the delivery efficiency of extrahepatic tissues such as muscles, fat, kidneys, nerves, eyes, and central nervous system is still insufficient, which remains a core weakness restricting the expansion of small nucleic acid drugs. In the next five years, delivery technology will enter the 2.0 era: new generation carriers such as antibody coupled nucleic acid (AOC), peptide coupled delivery systems, extracellular vesicle delivery, engineered vesicles, and novel degradable lipid nanoparticles (LNP) will gradually move from the laboratory to clinical validation. By accurately identifying specific receptors in different tissues, efficient targeted delivery to multiple organs and tissues throughout the body can be achieved, significantly increasing the concentration of drugs at the lesion site, reducing off target effects and systemic toxicity, and further opening up broader indications for tumors, neurodegenerative diseases, rare genetic diseases, autoimmune diseases, and more.
Multi functional small nucleic acid drugs have become a hot research and development topic, achieving synergistic treatment of “one drug, multiple effects”
Single gene silencing is no longer sufficient to meet the treatment needs of complex diseases, and in the future, small nucleic acid drugs will develop towards multifunctional and multi mechanism synergistic directions. On the one hand, the integration of gene silencing technology with tools such as gene editing and base editing can repair mutated genes while silencing pathogenic genes, achieving a dual effect of “inhibition+correction”; On the other hand, the combination of immune activation modules and targeted delivery systems enables small nucleic acid drugs to silence oncogenes or pathogenic genes while activating local immune responses, enhancing anti-tumor and antiviral effects. In addition, dual target and multi-target small nucleic acid drugs will be further popularized, improving efficacy and reducing drug resistance by synchronously regulating multiple disease pathways, truly realizing the new generation of drug design concept of “one drug, multiple effects, one drug, multiple indications”.
AI deeply empowers full chain research and development, significantly speeding up, reducing costs, and increasing efficiency
Artificial intelligence will fully penetrate the key links of small nucleic acid drug research and development, reshaping the industry’s research and development paradigm. In the sequence design stage, AI models can quickly screen the optimal sequence with high specificity, high stability, and low off target risk; In terms of chemical modification optimization, machine learning can accurately predict the impact of modification sites on half-life, immunogenicity, and delivery efficiency, reducing a large number of trial and error experiments; In the development of delivery systems, AI can simulate the interaction between carriers, cell membranes, and tissue microenvironments, improving the accuracy of delivery efficiency prediction. Overall, the large-scale application of AI will shorten the research and development cycle of small nucleic acid drugs by more than 50%, significantly improve the success rate of clinical translation, reduce research and development costs, promote more early pipelines to quickly enter the clinical stage, and accelerate the overall innovation pace of the industry.
summary
2026 is a crucial year for the outbreak of the small nucleic acid industry. Driven by breakthroughs in delivery technology, expansion of chronic disease indications, and capital policies, small nucleic acids have officially entered the billion dollar chronic disease market from the field of rare diseases, becoming the core force of the third-generation pharmaceutical revolution. In the next five years, with the upgrading of delivery technology, the development of multifunctional drugs, and the deep empowerment of AI, the small nucleic acid market will continue to grow rapidly, and China is expected to become the world’s most important growth engine. For enterprises, capital, and patients, the small nucleic acid track is both an innovation highland and a promising future. China’s biopharmaceutical innovation will also achieve leapfrog development through this.
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Disclaimer: This research report is based on the public market data, clinical research progress, enterprise dynamics, and industry policies of the small nucleic acid industry in 2026. It is only for industry reference and information sorting for practitioners and investors in the biopharmaceutical field, and does not constitute any investment advice, cooperation offer, or decision-making basis.
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