RNA interference (RNAi) is a natural gene-silencing mechanism triggered by double-stranded RNA (dsRNA) that can specifically inhibit the expression of target genes. Since Fire and Mello first discovered the RNAi mechanism in 1998, scientists have continuously delved into this process and gradually developed RNAi therapies. This therapy can target specific genes to prevent their overexpression or abnormal expression, thereby treating various diseases.
One of the key advantages of RNAi therapy is its high specificity and efficacy. By precisely designing small interfering RNA (siRNA) sequences, it is possible to effectively silence specific disease-related genes. This specificity makes RNAi therapy show great potential in treating genetic diseases and some specific types of cancers.
Unlike traditional drug therapies, RNAi therapy directly regulates gene expression rather than interfering with protein functions, allowing precise combating of diseases without harming other cells and genes. RNAi therapy has lower side effects, and due to the longer half-life of siRNA molecules in the body, patients need less frequent dosing, greatly improving patient compliance.
In 2018, the world's first siRNA therapeutic drug targeting the liver was approved, marking a significant breakthrough in the transition of RNAi therapy from the laboratory to the clinic. Since then, multiple siRNA drugs have been approved, primarily through binding with N-acetylgalactosamine (GalNAc) ligands to ensure precise delivery of siRNA to the liver. These therapies have already shown high efficacy and durability, gradually expanding from treating rare diseases to wider chronic disease areas.
With the continuous development of RNAi therapeutics, we expect to see its application in more fields in the future. RNAi not only has significant advantages in treating genetic diseases but will also achieve breakthroughs in metabolic, cardiovascular, immune-mediated related diseases, and more. Youngen is committed to promoting the widespread application of RNAi therapy, utilizing cutting-edge technology platforms to bring more treatment options to millions of patients worldwide, helping to improve their quality of life.
RNAi therapy represents a revolutionary advancement in the field of gene therapy. With its high specificity and low side effects, it is providing new hope for treating various complex diseases. As an industry leader, Youngen will continue to promote the research and application of RNAi therapy, developing more innovative treatments to offer better therapeutic options for patients around the world.
