British biotechnology company MiNA Therapeutics ‘innovative RNA therapy may enhance liver cancer patients’ response to standard treatment. The therapy uses a double-stranded RNA that can activate a target gene called CEBPA. Packaging double-stranded RNA in lipid nanoparticles helps to penetrate into liver cells that are often difficult to reach and can control gene expression in the nucleus. It is understood that the low level expression of certain genes is related to liver cancer and other liver diseases. In laboratory studies, increasing the expression of CEBPA to restore its protein levels to normal can help reduce the growth of cancer cells.
Among the patients receiving biotechnological small activated RNA (saRNA), two of them showed a complete response after receiving sorafenib, and the other showed a partial response after treatment with lenvatinib. This is the first trial of saRNA therapy in humans. Since the research is still in its early stages, biotech companies now hope to gather more relevant evidence.
The company also hopes to conduct the same drug test in patients with cirrhosis in the future, and also cooperates with Boehringer Ingelheim to carry out other projects for liver diseases. After a long period of development, more and more RNA treatments have entered the market. Unlike MiNA therapies that activate gene expression, most use RNA interference (RNAi) technology to reduce gene expression. Recently, the European Commission approved Onpattro, the first RNAi drug developed by Alnylam for the treatment of polyneuropathy.