Our Historyis closely tied to the C3P3 technology – the first ever non-viral artificial eukaryotic expression systemdeveloped from scratch by Eukarÿs
C3P3 artificial eukaryotic expression system synthesizes autonomously and at high yields the messenger RNA(s) of interest (mRNA) in the host cell cytoplasm, thereby leading to high production levels of the target protein(s).
The crucial role of mRNA in the flow of genetic information explains that the C3P3 system has a wide range of in vivo therapeutic and non-therapeutic applications in the entire eukaryotic world.
Until late 2000s, the development of an artificial eukaryotic expression system was nearly impossible due to the amazing complexity of eukaryotic post-transcriptional mRNA modifications.
The emergence of synthetic biology during the last decade was pivotal, allowing the construction of the C3P3 expression system (stands for chimeric cytoplasmic capping prone phage polymerase).
The development of the first generation of C3P3 (C3P3-G1) was completed early 2016 with good performances with CpG-free DNA templates, which are preferably used for our synthetic gene therapy – a novel therapeutic approach designed as safe, efficient and operating for most human disorders.
Eukarÿs is currently finalizing the third generation of its C3P3 system (C3P3-G3) with much greater complexity and performances than the initial prototype. C3P3-G3 is used for both synthetic gene therapy and bioproduction tools.
Today, Eukarÿs is a preclinical-stage biotechnology company that develops a pipeline of synthetic gene therapy treatments for liver diseases. Synthetic gene therapy is a radically novel therapeutic approach, which exploits the C3P3 system and is designed to be safe and efficient for the treatment of either rare monogenic diseases or frequent multifactorial complex disorders. The advanced prototype of EUK-LPR, our first synthetic gene therapy treatment, has demonstrated excellent performances in animal models. Further therapeutic programs will be launched in 2019.
Tomorrow, Eukarÿs will extend its synthetic gene therapy approach to other therapeutic areas taking advantage of its generic nature. The therapeutic areas to be addressed will be diseases associated with secreted protein deficiency and the field of immuno-oncology.
Besides its therapeutic development, Eukarÿs develops tools for the bioproduction of viruses and proteins, which also exploit the C3P3 artificial expression system. These tools have already shown excellent performances for the bioproduction of viruses and proteins. These tools will be available mid-2019 for research purposes.