Chinese Biotechs Surge into the Spotlight as Small Nucleic Acid Drugs Enter Boom Phase

　　【Pharmaceutical Network Market AnalysisSmall nucleic acid drugs are typically single-stranded or double-stranded DNA or RNA molecules, tens of base pairs in length, which primarily act on mRNA within cells through base pairing. By regulating protein expression, they achieve the goal of treating diseases. Compared with traditional chemical drugs and biologics, small nucleic acid drugs offer many advantages. Industry insiders indicate that small nucleic acid drugs are becoming the next breakthrough in China-produced innovative medicines.
 

Small nucleic acid drugs, as an emerging form of medication, have ushered in a promising start for China-produced small nucleic acid drugs in 2024. For instance, on January 3, Suzhou Ribo Life Science and Ribo International Research and Development Center reached a cooperation agreement with Boehringer Ingelheim regarding their innovative small nucleic acid therapy for treating non-alcoholic or metabolic dysfunction-associated steatohepatitis (MASH), with a total transaction value exceeding 2 billion US dollars. On January 7, Bowang Pharmaceutical announced that it had entered into two exclusive licensing cooperation agreements with Novartis for RNAi therapies. The potential total value of the two deals amounts to 4.165 billion US dollars.
 

It is reported that Ribo Life Science currently has 8 small nucleic acid drugs in clinical trials, with research pipelines covering chronic diseases, oncology, inflammation, and ophthalmology. Among them, 5 are based on the self-developed GalNAc (N-acetylgalactosamine, the most common conjugation system) small nucleic acid drug delivery technology platform, RIBO-GalSTARTM. In the field of liver diseases, RBD1016's two indications for hepatitis B and hepatitis C have entered phase 2 and phase 1 global clinical trials, respectively. An siRNA drug targeting Caspases 2 for optic nerve protection, RBD1007, is under development for the indication of non-arteritic anterior ischemic optic neuropathy (NAION) and has reached phase 3 clinical trials.
 

Bowang Pharma focuses on the development of siRNA drugs and has established multiple technology platforms and a rich R&D pipeline. It has developed product pipelines in the fields of cardiovascular diseases, rare diseases, viral infections, metabolic diseases, and central nervous system diseases. Among them, BW-01 and BW-02 in the cardiovascular field have entered the clinical stage and are highly likely to be the core products authorized to Novartis this time. According to the filing information, these two cardiovascular products are used for treating dyslipidemia and hypertension, respectively.
 

Data shows that small nucleic acid drugs are experiencing explosive growth. From 2016 to 2022, the global market size of small nucleic acid drugs surged from 0.01 billion USD to 3.8 billion USD, with a compound annual growth rate (CAGR) of 295%. Reportedly, the nearly 300% CAGR in market size has also attracted pharmaceutical companies such as Junshi Biosciences, Hengrui Pharma, Chengdu先导, and Yuekang Pharmaceuticals to accelerate their entry into the field.
 

In April 2023, Junshi Biosciences initiated a clinical trial for an ANGPTL3 siRNA drug, positioning itself as an innovative biotech company exploring small nucleic acid drugs. Hengrui Pharma also launched the clinical trial for its siRNA drug HRS-5635 in 2023. Other publicly listed companies such as Brii Biosciences, Yikang Pharmaceuticals, CSPC Pharmaceutical Group, and HitGen have also made significant advancements in the field of small nucleic acid drugs.
 

Small nucleic acid drugs have broad market prospects, and as companies actively lay out their strategies, the small nucleic acid drug market continues to awaken. According to data, as of the end of 2023, 19 small nucleic acid drugs have been approved globally, with treatment areas mainly concentrated in rare disease fields such as DMD, rare dyslipidemia, SMA, and ALS. In 2023, the FDA approved four small nucleic acid drugs for marketing: TOFERSEN (Amyotrophic Lateral Sclerosis) by Biogen/Ionis, EPLONTERSEN (Polyneuropathy) by AstraZeneca/Ionis, NEDOSIRAN SODIUM (Type 1 Primary Hyperoxaluria) by Novo Nordisk, and AVACINCAPTAD PEGOL SODIUM (Geographic Atrophy Secondary to Age-Related Macular Degeneration) by Astellas.
 

However, some experts pointed out that although small nucleic acid drugs have made significant progress in recent years, they were once restricted by nucleic acid delivery technology, leading to a bumpy development path. Around 2010, due to limitations in the delivery system, the research and development of small nucleic acid drugs faced repeated setbacks.
 

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