ABOUT OUR TECHNOLOGY
Proteins are the “workhorses” within cells responsible for metabolic processes, transport processes and structural elements. They are coded by genes on DNA and this code is transcribed into a message (messenger RNA or mRNA) within the cell nucleus. This mRNA then leaves the nucleus and is translated into the protein on ribosomes (Figure 1). Therapeutic mRNA is being developed as a new class of biological drug. Such therapeutic mRNA can address a wide range of different diseases as described below.
Although therapeutic mRNA has the potential to address a wide range of diseases, these biological drugs are very easily degraded in the body and are also large polar molecules which are not able to enter cells by themselves. They therefore require a carrier to protect them after administration and to allow delivery to the cell cytoplasm where they can then be translated into the corresponding therapeutic protein. Acuitas has developed LNP systems that can protect mRNA and very efficiently deliver these drugs to where they are needed in the body. In both preclinical and clinical studies, Acuitas LNP have been shown to be safe and effective