Small nucleic acid drugs were one of the focal points at this year's JPM conference.

Overseas giants Alnylam and Ionis have both announced breakthroughs in sales of their core products , reflecting the accelerated commercialization of the entire industry.

Meanwhile, Chinese companies are rapidly establishing their positions , with innovative biotechnology companies beginning to export their independently developed technologies and pipelines globally, actively participating in global competition. Large domestic pharmaceutical companies are also accelerating their expansion in this sector; China Biopharmaceutical's acquisition of Hegia for 1.2 billion yuan indicates that the small nucleic acid technology sector is moving from the periphery to the mainstream.

01

High-growth track
the market size of small nucleic acid drugs has grown rapidly with breakthroughs in the field of chronic diseases . According to a Frost & Sullivan report, the global small nucleic acid drug market size is expected to grow from US$5.7 billion in 2024 to US$20.6 billion in 2029.

The performance of leading companies has validated this prediction.

At the JPM conference, Alnylam identified 2025 as a turning point for the company, with its four self-developed commercialized siRNA drugs expected to generate nearly $3 billion in revenue in 2025, representing an 81% year-over-year increase and achieving non-GAAP profitability.

Sales of the TTR series products more than doubled to approximately $2.5 billion, and sales are projected to reach $4.4-4.7 billion by 2026, with a median growth rate of 83%.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative cardiomyopathy caused by the aggregation of misfolded transthyretin (TTR) monomers into amyloid fibers that deposit in the myocardial interstitium. This progressive restrictive cardiomyopathy leads to heart failure, conduction abnormalities, and arrhythmias, with high rates of disability and mortality. Once considered a rare disease, its detection rate in patients with heart failure is increasing annually as our understanding deepens.

Currently, the TTR drug market is dominated by Pfizer's TTR stabilizer Tafamidis (Vyndaqel/Vyndamax), with global sales exceeding $5 billion in 2024. This drug reduces cardiovascular mortality and hospitalization risk by inhibiting tetramer dissociation and amyloid fibrillation. It works similarly to "reinforcing a dam" to prevent flooding (misfolded proteins). However, it cannot eliminate existing proteins, nor can it completely prevent their formation.

Alnylam's Amvuttra (Vutrisiran) represents a completely new treatment logic—RNAi silencing agents. It acts on TTR mRNA within liver cells, directly degrading the mRNA using a RISC complex, thereby blocking TTR protein synthesis. This is equivalent to "cutting off the water source." Clinical data show that Vutrisiran has significant efficacy in improving neuropathy symptoms and reducing serum transthyretin (TTR) levels.

The drug was approved by the FDA in 2022 for the treatment of adult hereditary transthyretin-mediated (hATTR) amyloidosis polyneuropathy. In 2025, it was approved for a new indication for the treatment of wild-type or hereditary ATTR amyloid cardiomyopathy (ATTR-CM).

The approval of this new indication is based on the results of the Phase 3 Helios-B clinical trial, which showed that it reduces the risk of cardiovascular death/hospitalization and improves patients' quality of life . In the overall population, Amvuttra significantly reduced the risk of all-cause mortality and recurrent cardiovascular events by 28%. Furthermore, compared to the daily oral administration of Tafamidis, Amvuttra only requires subcutaneous injection once every 12 weeks.

These data suggest that Amvuttra has the potential to challenge Tafamidis's position as the next generation of standard TTR therapy and propel Alnylam into a new phase of profitability.

Another leading small nucleic acid company, Ionis, also ushered in a key turning point in 2025.

In 2025, it launched its first self-developed commercial product, Tryngolza (olezarsen), which generated $105 million in sales revenue and is expected to become its first blockbuster drug.

This drug is a first-in-class antisense oligonucleotide (ASO) therapy developed by Ionis, which regulates triglyceride metabolism in the blood by inhibiting the body's production of apolipoprotein C-III (apoC-III).

In December 2024, olezarsen was approved by the U.S. FDA as the first treatment for familial chylomicronemia syndrome (FCS), administered via an auto-injector once a month.

However, the drug has greater potential for use in the indication of severe hypertriglyceridemia (sHTG).

In September 2025, olezarsen achieved positive topline results in the pivotal Phase 3 CORE and CORE2 studies in sHTG patients. These two pivotal trials demonstrated that after 6 months of treatment with 80 mg olezarsen, patients experienced a 72% reduction in triglyceride levels (adjusted for placebo), and olezarsen also reduced the risk of acute pancreatitis (AP) by 85%, setting a record as the first and only study in this field to achieve this efficacy.

Ionis has submitted a marketing application for Tryngolza for sHTG, and if it is granted priority review status, it is expected to be approved for this indication this year.

Based on strong clinical data, Ionis has raised its peak sales guidance for Tryngolza from "over $1 billion" to "over $2 billion".

02

Chinese pharmaceutical companies are quietly positioning themselves.

Against the backdrop of the rapid development of the small nucleic acid field, Chinese pharmaceutical companies are quietly positioning themselves, participating in global competition with unique technological approaches and differentiated layout strategies.

According to data from PharmNet, dozens of small nucleic acid drugs in China have entered the clinical research stage. Meanwhile, companies such as Bowang Pharmaceutical and Ribobio have already partnered with multinational corporations (MNCs) to participate in international competition.

Among the small nucleic acid companies that showcased themselves at the JPM conference were Chinese pharmaceutical companies.

Yaojing Gene has released its independently developed Kardia Shuttle small nucleic acid cardiac targeted delivery platform technology, filling the global gap in cardiac targeted siRNA therapy.

This platform, leveraging the TA-SEEK™AI large-scale model, has discovered the world's first target that is specifically and highly expressed on the surface of cardiomyocyte membranes, enabling precise targeted delivery of multiple drugs, including siRNA and ASO, to cardiac tissue. Preclinical studies have shown that its targeting, activity, and durability are superior to or comparable to the current benchmark liver-targeting technology, GalNAc, and its efficacy has been validated from mice to large animals, marking a substantial step forward in cardiac-targeted siRNA therapy.

Argo Biopharma, which has been twice "rebranded" by pharmaceutical giant Novartis , also attended this year's JPM conference and shared the company's latest business progress and outlook.

Leveraging its liver-targeting and extrahepatic delivery technology platform, the company has built a diversified RNAi drug development pipeline covering cardiovascular metabolism, special diseases, viral infections, central nervous system diseases, and rare diseases. Currently, six RNAi drug candidates have entered clinical trials.

In January 2024, Bovoi Pharmaceuticals signed a collaboration agreement with Novartis to jointly develop multiple innovative siRNA drugs for cardiovascular diseases. Bovoi Pharmaceuticals received an upfront payment of US$185 million, with a potential total collaboration value of up to US$4.165 billion.

In September 2025, Bowen Pharmaceuticals once again entered into a collaboration with Novartis on multiple cardiovascular products, receiving an upfront payment of $160 million and a total potential milestone value of up to $5.2 billion.

Meanwhile, the small nucleic acid sector is booming in the domestic financing market.

RiboBio recently listed on the Hong Kong Stock Exchange, raising a total of HK$1.593 billion. Leveraging its independently developed RiboGalSTAR liver-targeted delivery platform, the company has established a robust pipeline of siRNA drugs, with seven in clinical trials and four having entered Phase II clinical trials.

In terms of business development, in January 2024, Ribobio entered into a collaboration with Boehringer Ingelheim to jointly develop innovative small nucleic acid therapies for the treatment of non-alcoholic or metabolic disorder-related steatohepatitis, with a total transaction value exceeding US$2 billion.

In December 2025, SanegeneBio announced that it had completed a Series B financing of over US$110 million, which will be used to leverage the company's globally leading RNAi drug development platform, including the LEAD extrahepatic delivery platform, to advance its clinical-stage pipeline into registration studies and accelerate the development of its pipeline in multiple therapeutic areas.

Prior to this, St. Johns Hopkins Biotech and Eli Lilly and Company entered into a global research and development collaboration and licensing agreement, under which the two parties will jointly promote the development of RNAi drug candidates for metabolic diseases based on St. Johns Hopkins Biotech's proprietary LEAD platform.

Large domestic pharmaceutical companies are also accelerating their expansion into this sector.

On January 13, 2026, China Biopharmaceutical announced that it would acquire Hergia, a pioneering siRNA biopharmaceutical company, for a total price of RMB 1.2 billion.

Data shows that Hergia has 6 intrahepatic/extrahepatic delivery platforms, 4 innovative drugs in clinical trials, more than 20 projects in preclinical stages, and multiple pipelines with global FIC and BIC potential, covering the three major chronic disease treatment areas of weight loss and metabolism, cardiovascular and cerebrovascular diseases, and nervous system diseases.

03

Conclusion

With breakthroughs in delivery technology, small nucleic acid drugs are showing transformative potential in the treatment of chronic diseases such as cardiovascular and metabolic disorders. From Novartis' Leqvio to Alnylam's Amvuttra and Ionis' Tryngolza—the successive emergence and commercial success of these blockbuster drugs signify that small nucleic acid therapies have moved from scientific concepts into the mainstream treatment landscape.

While no domestically produced small nucleic acid drugs have yet been approved for marketing in China, a number of companies have gradually expanded overseas through technology licensing (BD), indicating the rise of Chinese power.

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