Before 2020, nucleic acid-based microscopic research primarily contributed to understanding disease mechanisms and diagnostics. However, after 2020, nucleic acid-based drugs began to be rapidly applied in disease treatment.
For example, in 2021, mRNA vaccines were officially approved. This novel vaccine mechanism, characterized by fast development, high safety, and significant effectiveness, attracted much attention. In 2023, two scientists who made outstanding contributions to this technology were awarded the Nobel Prize in Medicine. The rapid progress—from large-scale clinical application to award recognition in just two years—is rare in Nobel Prize history.
Another example is Inclisiran, a long-acting lipid-lowering injection, which was approved for use in the United States in December 2021. Dubbed the “cholesterol-lowering vaccine,” it was approved by China’s National Medical Products Administration (NMPA) in August 2023, offering a new medication option for patients with hyperlipidemia.
Furthermore, in 2024, a long-acting antihypertensive drug, Zilebesiran, completed its Phase II clinical trials. The results showed that patients only need to inject the drug twice a year to maintain long-term blood pressure stability. If all goes smoothly, Zilebesiran may be officially launched in 2026.
Although these new drugs treat different diseases, they all share one significant feature: their main components are nucleic acids, and their mechanisms of action regulate protein synthesis at the microscopic level.
The emergence of these nucleic acid-based drugs is disrupting our traditional understanding of disease treatment and is bringing about an interesting shift, which I would summarize as: “Future drugs will not look like drugs, and patients will not look like patients.”
Why Future Drugs Will Not Look Like Drugs
Next, using Inclisiran, a new lipid-lowering drug, as an example, I will explain why the application of such new drugs will bring disruptive changes to disease treatment.
Before we begin, let’s briefly introduce the basic concept of lipid-lowering drugs. When we talk about “lowering lipids,” we mostly refer to reducing low-density lipoprotein cholesterol (LDL-C), also known as “bad cholesterol.”
LDL-C is considered a major culprit in cardiovascular and cerebrovascular diseases. Studies show that among many modifiable risk factors for cardiovascular and cerebrovascular diseases, lowering LDL-C is an important and effective intervention. This is mainly because it reduces vascular inflammation, protects vascular health, and decreases the incidence of adverse events such as cardiovascular diseases. Inclisiran is an effective lipid-lowering drug that reduces LDL-C levels.
The reason I believe future drugs will not look like traditional drugs starts with their composition, which is different from that of traditional drugs.
Traditionally, we believe that drugs mainly exist as chemical substances that intervene in a specific physiological process of a disease, thereby interrupting the causal chain of the disease’s progression to achieve therapeutic effects. For example, traditional lipid-lowering drugs, such as statins (e.g., atorvastatin, simvastatin), are small-molecule compounds. Once these drugs enter the cells, they inhibit the activity of an enzyme responsible for synthesizing LDL-C, thus reducing its production and lowering cholesterol levels.
However, the newly launched Inclisiran differs from traditional drugs. Its active ingredient is not a chemical substance, but a piece of nucleic acid, which you can think of as a “code” that influences and controls protein synthesis. Once this nucleic acid enters the human body, it directs the body to produce proteins that have therapeutic effects. For example, the mRNA COVID-19 vaccine directs the body’s cells to produce a protein called the “S protein,” which, once inside the body, stimulates the immune system to help the body fight the virus. This is the basic principle of mRNA vaccines.
In addition to creating proteins, nucleic acid drugs can also interfere with protein synthesis. For instance, the active ingredient in Inclisiran is a special molecule that, once inside the cell, interferes with signal transmission. These signals would normally instruct the cell to produce a protein that increases blood lipid levels. By blocking these signals, Inclisiran effectively reduces the production of this protein, ultimately lowering LDL-C levels, or “bad cholesterol.”
This is the first point: the composition of new drugs is different from traditional drugs.
“Drugs don’t look like drugs” also refers to a breakthrough in the drug development concept, which is the second point.
The core of traditional drug development is to find chemical substances that can inhibit disease symptoms. However, in the era of nucleic acid drugs, the development concept has shifted—researchers no longer need to screen through complex chemical substances. Instead, their role is more like that of a programmer: they simply need to understand which protein needs to be regulated for a specific disease and then encode the nucleic acid sequence that regulates that protein.
When nucleic acid drugs enter the body, they either help the body produce new proteins or reduce the synthesis of disease-related proteins. This entirely new model will significantly change the treatment paradigm for many chronic diseases. In the future, many diseases caused by abnormal proteins may have more effective treatments through nucleic acid drugs.
For example, hemophilia is a genetic disorder in which patients are unable to synthesize critical proteins—specifically, certain clotting factors. If we can inject the nucleic acid that guides the synthesis of these clotting factors into the patient’s body, instructing the body to produce the missing clotting factors, we could potentially treat hemophilia. This method may very well become an important treatment option for hemophilia patients in the future.
Similarly, diseases like rheumatoid arthritis, ankylosing spondylitis, colitis, lupus, and psoriasis, all related to autoimmune disorders, are often stubborn and difficult to treat. Their mechanisms are often linked to the abnormal expression of certain inflammatory factors. If nucleic acid drugs can guide the body’s cells to produce proteins that counteract these inflammatory factors, they could effectively suppress excessive immune responses and treat these chronic diseases.
In other words, many disease treatment models will be completely revolutionized by nucleic acid drugs. This is the second point.
The third point, “drugs don’t look like drugs,” also applies to the major changes in drug administration methods.
The frequency with which we take a drug depends on the drug’s metabolic speed in the body. Most traditional drugs have a fast metabolic speed and a short half-life, so patients need to take them frequently. For example, traditional statin drugs typically need to be taken daily. But new nucleic acid drugs, like Inclisiran, have a long-lasting effect and only require injections every six months. This is because they use a slow-release system that allows the drug to be gradually released inside the cells, significantly extending its half-life.
As mentioned earlier, this long-acting injection is sometimes referred to as a “cholesterol-lowering vaccine.” Calling it a “vaccine” doesn’t mean it prevents high cholesterol, but rather that it has such a long half-life that one injection can provide long-lasting therapeutic effects, similar to a vaccine. This administration method significantly improves patient compliance and reduces the risk of treatment failure due to missed doses, helping to manage blood lipids over the long term.
Currently, this new lipid-lowering drug is typically used in combination with traditional medications or as a substitute when patients cannot tolerate traditional drugs or experience severe side effects. Nevertheless, it undoubtedly provides new options and hope for patients needing lipid-lowering treatment.
Why Patients Don’t Look Like Patients
As we mentioned earlier, nucleic acid drugs are completely different from traditional drugs in terms of their composition, mechanism of action, and even administration methods. These changes also make patients “not look like patients.”
“Patients don’t look like patients” actually refers to the change in the role of patients. In traditional views, patients are often bound by their diseases, needing long-term medication and constant attention to their condition, while enduring both physical and psychological pressure. However, as we mentioned earlier, new drugs like long-acting lipid-lowering injections only need to be administered once every six months, so patients no longer have to remember to take medicine every day, freeing them from constant attention to their condition. This entirely new treatment method significantly reduces the interference of diseases in the patient’s daily life, allowing them to enjoy more freedom.
New drugs also have a positive impact on the patient’s psychological state. Simplifying medication regimens reduces the burden of complicated drug-taking processes, which in turn alleviates psychological stress. This sense of relaxation can improve the patient’s quality of life and increase their confidence in overcoming the disease. At the same time, the personalized treatment approach enables patients to actively participate in the treatment process, collaborating with doctors to develop treatment plans suited to their needs, leading to more efficient disease management.
“Patients don’t look like patients” is not only meaningful for individual patients, but it also brings positive impacts to society as a whole. As more new treatments emerge, patients can recover more quickly and return to their normal work and life, creating more value for society. Of course, this also reduces the burden on the healthcare system and improves the efficiency of healthcare resource utilization.
Conclusion
This is the new change brought by nucleic acid drugs—”drugs don’t look like drugs, and patients don’t look like patients.”
Undoubtedly, the success of these new drugs is the result of breakthroughs in microscopic research, which have laid a solid foundation for a deeper understanding of diseases and treatment strategies on a macro level. The trend represented by “drugs don’t look like drugs, and patients don’t look like patients” brings new hope and motivation to medicine.
To summarize:
First, the success of nucleic acid drugs further proves the importance of studying diseases at the cellular and molecular levels, and the crucial role microscopic research plays in advancing medical progress. Through this research method, humanity can not only gain a more comprehensive understanding of the nature of diseases, but also discover more potential therapeutic targets. It lays a solid foundation
for breakthroughs in new therapies.
Second, breakthroughs in the era of nucleic acid drugs will change disease treatment concepts. We will no longer focus on chemical substance screening, but more on encoding specific proteins to regulate or suppress disease progression. The treatment models of chronic diseases will be revolutionized.
Finally, new nucleic acid drugs may lead to patient treatment experiences that go beyond our imagination, offering a more convenient and patient-centered healthcare environment.
