Nucleic acid drugs will bring about a turning point in the treatment of non-alcoholic fatty liver disease and acute respiratory distress syndrome

Source: Learning to Build a Strong Country  核酸药物将使非酒精性脂肪性肝病和急性呼吸窘迫综合征的治疗出现转机 (

With the success of mRNA COVID-19 vaccine, nucleic acid drug research has once again attracted the attention of biomedical scientists around the world. In Guangzhou, there is a nucleic acid Drug development platform that was established two years ago and has the first independent intellectual property rights in China. It is quietly carrying out research on nucleic acid drugs, the top technology of biomedicine, and actively exploring key core technologies to further integrate the pharmaceutical industry resources of the Guangdong–Hong Kong–Macao Greater Bay Area, and walk out a "Guangzhou path" for high-quality development of the biomedicine industry.


Autonomous nucleic acid drugs to fill clinical needs

Nucleic acid drugs are gradually becoming a focus of biopharmaceutical investment and a hot topic for pharmaceutical enterprise research and development. More than 80% of the pathogenic proteins related to human diseases cannot be targeted by conventional small molecule drugs and biomacromolecule preparations, and are considered non drug protein targets. RNA (ribonucleic acid) is an important bridge connecting genes and proteins, so nucleic acid drugs are not only not limited by their own structure, but can break the "difficult to form drugs"; It can also significantly expand the target range, break the "non producibility", and compare with traditional drugs, its efficacy is more powerful and long-lasting. Nucleic acid drugs are recognized as "sharp weapons" against cancer, Alzheimer's disease, diabetes and other persistent diseases, and are also the hot frontier of global new drug research and development. At present, the vaccination of hundreds of millions of doses of mRNA COVID-19 vaccine around the world has verified its effectiveness, and also made the overall nucleic acid Drug development fever rise again.

At present, 14 nucleic acid drugs have been launched globally and positive market feedback has been received, indicating the booming development of the nucleic acid drug industry. Due to the large number of patients in China and the large market development space, in the future, with the improvement of research and development capabilities of small nucleic acid drug development enterprises in China, the small nucleic acid drug market is expected to usher in rapid development, gradually entering a stage of differentiated innovation and breakthrough innovation, especially for drugs with larger indications for patients, such as potential therapeutic drugs for hepatitis B, which will further drive the rapid development of the market.

China has a special position in the field of nucleic acid Drug development. For the two Danish scientists, the feeling is still profound. As the owner of Blockmir, an independent intellectual property research and development platform for nucleic acid drugs, Thorleif M ø ller, a Danish biopharmaceutical expert, is introducing him to Huangpu District, Guangzhou through the national talent introduction plan and starting to deepen his research and development in Guangzhou's biopharmaceutical industry. Thorleif Moller and his partner Christina Udersen both believe that in many technological fields such as electric vehicles, smartphones, batteries, etc., the Chinese market is the fastest-growing market in the world.

Although Denmark is strong in life science research, it is only a small market and country with limited patients and funding. Although China is still catching up in nucleic acid Drug development technology, it is a huge country and market. "Especially the Chinese investors are really far sighted and can see the great future. We have no experience in dealing with China before. So this is a new world for us. Indeed, when people enter the new Universal Time, they will be a little worried and afraid, but our cooperation with the Chinese people is the best experience so far. We have been open, friendly and a lot of trust.".

Advanced technology leads multi-party cooperation and escort

Thorleif Moller admits that the main achievement in the development of nucleic acid drugs in recent years has certainly been mRNA nucleic acid vaccines. This is a product developed in a record time and a major commercial success for nucleic acid drugs, which has attracted more attention from the world for nucleic acid drug research. "Perhaps it can really help us out of this epidemic.

But the research on nucleic acid drugs will still accelerate the progress of disease research that covers a wider population. "For example, Central nervous system disease, Respiratory disease and nonalcoholic fatty liver diseases brought about by COVID-19, etc. In these fields with a wide range of patients, we strive to surpass from the perspective of nucleic acid drugs, and also find a more direct relationship between genes and diseases through the continuous advancement of technology platforms, so that drugs can be more easily recognized and developed. At the same time, we also pay attention to connecting external cooperation forces to jointly reduce risks to the maximum Low. He further explained that the basic concept of nucleic acid drugs is to go beyond traditional drugs. Traditional drugs are small molecules and antibodies, targeting proteins, but only a small portion of proteins can be targeted by small molecules. Our technical means will expand the target space while finding more targets.

Christina Udersen, on the other hand, stated that in drug development, researchers generally rarely share research results with their peers. But Christina Udersen believes that the nucleic acid drug industry is very unique. "It is like a community that chooses to be open to scientists within the industry and also participates in establishing academic collaborations, creating a very strong foundation for all companies working in this field because everyone can learn from each other.

For Christina Udersen, this also means that there are many things that can be predicted when researchers enter clinical trials. Because the chemical composition is more or less the same, the side effects of most nucleic acid drugs are very similar. That's why we can focus more on the actual sequence that depends on the target mechanism, rather than having to focus on the common toxicity of nucleic acid drugs. This is also why we can omit some steps when conducting clinical trials, as we have learned a lot about common chemical categories of nucleic acid drugs from platforms and the "community".

For example, the nucleic acid drug research for acute respiratory distress syndrome (ARDS), which is currently being carried out in cooperation with the First Affiliated Hospital of Sun Yat-sen University, has entered animal experiments, and the data has been positive. Data shows that by 2027, the global market of medical devices related to acute respiratory distress syndrome is expected to reach 16.9 billion US dollars, with a compound annual growth rate of 7.2% in the forecast period. This is driven by the increasing incidence rate and incidence rate of acute lung injury and related clinical conditions, the aging population and the lack of drug treatment.

Susceptible clinical factors include sepsis, pneumonia, inhalation trauma, pancreatitis, blood transfusion, and inhalation of smoke or toxic gases. Research on nucleic acid drugs for non alcoholic fatty liver disease (NASH) has also been prepared for animal experiments. With the improvement of people's living standards