Acuitas Therapeutics products

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.

Acuitas scientists, in collaboration with other researchers, have established the mechanism whereby certain LNP carriers can provide highly efficient delivery to hepatocytes in the liver (Akinc et al., Mol Ther. 2010). After intravenous administration, the PEG-lipid component that shields the surface of our LNP exchanges off. This allows binding of the plasma protein ApoE to the exposed neutral LNP surface. In the liver, the LNP with bound ApoE passes out of the blood vessels through holes (“fenestrations”) and accesses the interstitial space. The bound ApoE is then recognized by receptors on the surface of hepatocytes resulting in an uptake of the LNP into endosomes.