On January 9, 2026, China’s small nucleic acid drug R&D company Ribobio listed in Hong Kong. It surged 29.38% at the open, and its self-developed GalNAc delivery technology became the focus of market attention. The listing bell at the Hong Kong Stock Exchange seems to be beating the drums and clearing the way for the entire small nucleic acid drug track; a new round of pharmaceutical revolution has already raised its curtain.
Therapeutic areas: from rare diseases to chronic diseases
When traditional therapies are powerless against certain diseases, small nucleic acid (RNAi) drugs have demonstrated remarkable advantages. From targeting rare diseases to chronic common diseases, these “gene scissors” are showing their transformative potential across multiple therapeutic areas.
The world’s first siRNA drug patisiran was approved by the U.S. FDA in 2018 for the treatment of hereditary TTR amyloid polyneuropathy. Previously, patients with this disease had an average survival time of only 6–12 years, and the only option was liver transplantation. In the Phase III clinical trial, patisiran improved the neuropathy impairment score in 56% of patients, while the placebo group improved in only 4%. Its key advantage lies in precisely knocking down the pathogenic gene; after a single intravenous administration, the effect can last for weeks to months, greatly outperforming the short-lived effects of traditional supportive treatment.
Donidalorsen was approved in the United States in August 2025 for preventing attacks of hereditary angioedema. According to Phase III clinical data, the drug can reduce patients’ average monthly attack rate by as much as 97%, and it requires only one subcutaneous injection every 4 or 8 weeks. Compared with traditional therapies such as danazol, which may cause liver damage, or tranexamic acid, which increases thrombosis risk, Donidalorsen offers a more precise long-term management option with better safety.
Plozasiran was approved in China in January 2026 for the treatment of FCS (familial chylomicronemia syndrome). Patients with this rare hyperlipidemia face an acute pancreatitis mortality rate as high as 30%. Clinical trials show that the drug can reduce patients’ fasting triglyceride levels by 80% versus baseline, and likewise reduce the risk of acute pancreatitis occurrence by 80%. The convenient regimen of only four doses per year significantly improves patient adherence.
Inclisiran expands RNAi therapy into the cardiovascular disease field, which has a massive patient base. This siRNA drug targeting PCSK9 requires only two subcutaneous injections per year to continuously lower low-density lipoprotein cholesterol. Compared with PCSK9 monoclonal antibodies that require injections every two weeks or every month, Inclisiran’s dosing convenience gives it a clear advantage in improving patient adherence. This marks that small nucleic acid drugs are shifting from rare diseases toward common chronic disease areas that benefit a much broader patient population.
Treatment costs: high value, high pricing
From rare diseases to chronic diseases, small nucleic acid drugs are reshaping clinical practice with their breakthrough efficacy, but the economic considerations that follow have also become an unavoidable topic.
Inclisiran’s economic profile has been evaluated across multiple healthcare systems. In North America, the price per prefilled syringe of Inclisiran is USD 2,839.28. Based on the standard dosing regimen, the patient’s annual treatment cost is about USD 5,679. Compared with PCSK9 monoclonal antibodies that require injections once every two weeks and cost about USD 5,800–14,500 per year, Inclisiran maintains similar efficacy while reducing annual treatment costs by lowering dosing frequency.
Behind the high pricing of these drugs is an assessment of their clinical value and the benefits of long-term disease management. Taking Inclisiran as an example, its cost-effectiveness analysis is based on the expectation that it can reduce cardiovascular event risk by up to 26%.
Although definitive data must ultimately await the large cardiovascular outcomes study in 2026, the medication convenience brought by long-acting effects and the potential savings in medical resources have already made it an important option in the lipid management field.
It is worth noting that as more competitors enter the market and manufacturing processes mature, the long-term price trend of small nucleic acid drugs is expected to gradually decline. After Inclisiran entered China, it was priced at RMB 9,988 (USD value not provided in the original text). Based on 2 syringes per year, the annual treatment cost is about RMB 20,000 (USD value not provided in the original text). The drug was officially included in China’s National Reimbursement Drug List on January 1, 2026, which means patients’ actual out-of-pocket costs will drop substantially, significantly improving the accessibility of this long-acting therapy.
The short interval from Plozasiran receiving U.S. Breakthrough Therapy designation to being approved in China also reflects the urgency of global clinical demand for small nucleic acid drugs, as well as countries’ efforts to accelerate access to innovative drugs.
Competitive landscape: Still a blue ocean
The competitive landscape of the small nucleic acid drug market shows diversified characteristics. There are biotech companies that have focused on this field for decades, and there are also multinational pharmaceutical giants that are positioning themselves through cooperation, licensing, or acquisitions.
The table below summarizes the positioning of major global biotech manufacturers in the small nucleic acid drug field:
| Company name | RNAi field role / positioning | Key pipeline |
| Alnylam | RNAi therapy pioneer and leader | Has multiple marketed siRNA drugs (such as patisiran). Its ATTR therapy Vutrisiran is expected to see sales exceed USD 2.0 billion in 2025. |
| Ionis | Pioneer and leader in the ASO (antisense oligonucleotide) field | Seven ASO drugs have already been approved and marketed (including Donidalorsen), and it cooperates with multiple major pharmaceutical companies for development and commercialization. |
| Arrowhead | Focused on extrahepatic delivery technology platforms | Leverages the TRiM extrahepatic delivery platform to lay out weight-loss and CNS projects. Its product Plozasiran for treating FCS was approved for marketing in 2025. |
| Ribobio | Benchmark enterprise in China’s small nucleic acid drug field | Has self-developed GalNAc delivery technology and a liver-targeted delivery platform. Seven self-developed siRNA candidate drugs have entered the clinical trial stage. Core products: RBD4059 for treating FXI and RBD5044 for treating HTG. |
It is worth noting that pharmaceutical giants such as Novartis, Sanofi, and Pfizer are also actively building RNAi pipelines, among which Novartis has undoubtedly placed its bets the fastest, with frequent moves since 2019:
| Collaboration / acquisition target | Transaction amount and core content |
|---|---|
| Bowang Pharmaceuticals | Two collaborations in January 2024 and September 2025; the potential total amount is as high as USD 9.4 billion, jointly developing multiple siRNA drugs targeting cardiovascular diseases. |
| Arrowhead | In September 2025, paid a USD 200 million upfront payment and up to USD 2.0 billion in milestone payments, obtaining global authorization for the siRNA therapy ARO-SNCA for the treatment of Parkinson’s disease. |
| Other acquisitions | 2019: Acquired The Medicines Company for USD 9.7 billion, obtaining Inclisiran. |
| Other acquisitions | 2023: Acquired DTx Pharma for USD 1.0 billion, obtaining neural delivery technology. |
| Other acquisitions | 2025: Acquired Regulus for USD 1.7 billion, obtaining kidney disease therapies. |
Technical breakthrough: Delivery technology breakthroughs expand therapeutic boundaries
For small nucleic acid drugs to achieve broad application, they must overcome the challenges of delivery systems. As the easiest organ to target, the liver has already become the “main battlefield” of most small nucleic acid drugs, but scientists’ goals go far beyond that.
Ribobio’s self-developed GalNAc delivery technology is like installing a GPS navigation system on the drug, enabling it to precisely deliver the drug to liver target cells. But the current research focus has shifted to more difficult targets—such as the central nervous system and muscle tissue.
Competition in this field has expanded to the delivery platform level. Arrowhead’s TRiM platform is attempting to achieve targeted delivery to extrahepatic tissues, laying out projects in weight loss and central nervous system diseases.
Meanwhile, ADC technology that conjugates oligonucleotides with antibodies or peptide segments has emerged. This “precision-guided” strategy can deliver drugs specifically to tissues that are traditionally difficult to target, such as muscle. For example, an antibody–oligonucleotide conjugate therapy targeting Duchenne muscular dystrophy has already shown “unprecedented” protein expression capability in clinical trials.
Professor Robert Langer, an expert in drug delivery, once pointed out that the conjugation of cell/tissue-targeting ligands with oligonucleotide drugs is key to improving delivery efficiency and overcoming refractory diseases.
As these technologies mature, small nucleic acid drugs are expected to break through liver limitations and open up new battlefields in areas such as neurodegenerative diseases and muscle diseases.
Competition in the RNAi track has long since surpassed a contest of a single pipeline and entered a “three-part ensemble” stage of “platform defining potential, clinical validation proving strength, and commercialization realizing value.” The listings of Chinese companies such as Ribobio mean that we have already moved from the audience seats to the starting line of the main track. In this competition for the future of the pharmaceutical industry, Chinese companies are moving into the center of the stage.
[Disclaimer]: The above content reflects analysis of publicly available information, expert insights, and BCC research. It does not constitute investment advice. BCC is not responsible for any losses resulting from reliance on the views expressed herein. Investors should exercise caution.