In just four years, Shanghai-based Argo Biopharma has gone from a start-up to one of China’s most internationally recognised RNA innovators and has signed two major licensing deals with Novartis worth up to USD 6 billion. These Novartis deals could come to be one of the largest ever tie-ups between a Chinese biotech and a global pharma, representing validation of Argo’s siRNA (small interfering RNA/RNA interference (RNAi)) platform.
However, the origins of Argo’s story date back long before 2021. Co-founders Dongxu Shu (now CEO) and Patrick Shao (chief scientific officer) were both senior scientists at US-based Arrowhead Pharmaceuticals, a publicly traded firm and one of the best-known global biotechs in the RNA space.
Returning to China and setting out with an RNA play of their own from Shanghai, Shu and Shao are aiming to develop a new generation of therapies that overcome the durability, delivery, and safety limitations seen in earlier RNA drugs. Its proprietary RADS platform optimises siRNA chemistry to extend drug half-life and enhance liver delivery, addressing the short-acting limitations of first-generation RNA drugs.
Additionally – and in line with the work ongoing at more established companies – Argo has been exploring options for using RNA therapies to treat mainstream illnesses like heart disease and hypertension, as opposed to the niche conditions it has traditionally been used to target. Today, the company boasts a highly diversified clinical pipeline across rare diseases, viral infections, metabolic disease, CNS, heart, muscle, and obesity. Argo has advanced five programmes into the clinic, including two Phase II candidates in hypertriglyceridemia and hepatitis B.
RNA Explained
RNA therapies offer a way to tweak gene activity temporarily and precisely, helping treat diseases at their source without altering DNA permanently. The messenger RNA COVID-19 vaccines developed by Moderna and BioNTech have shown how this technology works in practice, as have RNAi drugs developed by the likes of Alnylam (metabolic, rare liver diseases, and cardiovascular conditions) and Ionis (neurological and cardiometabolic diseases). Several of these companies talk of RNA as the “fourth pillar” of therapeutics (after small molecules, antibodies/biologics, and cell/gene therapy).
In financial terms, RNA is already big business and is set to get even bigger. The global RNA therapeutics market stands at around USD seven to 12 billion today, depending on the inclusion of vaccines and newer modalities, with several forecasts predicting a market of tens of billions of USD by the early 2030s. Spinraza, an RNA therapy for the rare condition spinal muscular atrophy (SMA) developed by Ionis and marketed by Biogen, is now a blockbuster, generating USD 1.6 billion in global sales annually.
Argo has already raised over USD 100 million from primarily Chinese investors, but following the trend of global pharma tapping up Chinese biotechs for the rights to develop and commercialise early-stage medicines outside China, Argo has recently struck two deals with Novartis.
The most recent deal, in September 2025, saw Argo receive an upfront payment of USD 160 million, potentially rising to USD 5.2 billion, dependent on milestones and royalties, for the ex-China commercialisation rights of a heart disease drug. The 2024 deal between the two companies was potentially worth over USD four billion.
CEO Shu called the initial deal the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company” and added that the 2025 tie-up “further supports the innovation engine Argo has built to deliver best-in-class siRNA therapeutics while building a top-tier clinical development team across multiple geographies.”
Shaun Coughlin, global head of cardiovascular and metabolism at Novartis Biomedical Research said Argo’s candidates represent “an important paradigm shift in the prevention and treatment of cardiovascular diseases.”
A long and challenging journey lies ahead for Argo and its partner, Novartis. Yet, true to the spirit of the mythological ship it is named after, Argo’s leadership remains determined to navigate the obstacles ahead and, like Jason and the Argonauts, claim their own Golden Fleece: the successful commercialisation of their therapies and a leading place in the new era of RNA innovation.
