SamRNA, the future of RNA medicines
GOAL
Our goal is to facilitate and support the research and development of self-amplifying mRNA (samRNA) technologies. SamRNA is a designed RNA/mRNA platform, of which the core RNA/mRNA sequence is flanked with at least one RNA-dependent-RNA-polymerase (RdRP)-binding site in its 5’-end and 3’-end, respectively. The RdRP-binding sites can be recognized by viral replicase/RdRP enzymes, so as to activate RdRP-mediated cycling amplification of the designed samRNA in vitro, ex vivo as well as in vivo. By LNP-mediated delivery of both samRNA and RdRP mRNA into targeted cells, samRNA can be used as a safer and more effective RNA/mRNA medicine and/or vaccine to reduce administration dose, prevent potential side effects, and increase desired drug effects in the treated cells.
KEY TECHNOLOGY
Our research team has identified and further modified the coronaviral RdRP-binding sites, of which the related patents have been filed for not only securing world-wide IP protection but also supporting the new development of modern non-replicon-based samRNA technologies (the third generation self-amplifying RNA platforms). Two major samRNA technologies have been well established; one is Replicase/RdRP Cycling Reaction (RCR) and the other is samRNA medicine/vaccine.
* RCR is a novel in-vitro samRNA amplification technology optimized for industrial grade production of RNA/mRNA-based medicines as well as vaccines. Using recombinant viral replicase/RdRP enzymes, samRNA can be directly amplified from itself as a template without any hassle of DNA contamination. For preferential strand amplification in RCR, the desired sense (+) or antisense (-) strand samRNAs can be selectively amplified by using different combinations of strong and weak RdRP-binding sites in the designed samRNA construct. Most importantly, the amplified samRNA products are all in full-length conformation because both of its 5’- and 3’-end RdRP-binding sites must be intact in order to maintain the RCR activity, resulting in no or very limited RNA/mRNA degradation.
* SamRNA is the most advanced and advantageous RNA/mRNA medicine platform in the world. By changing the middle core RNA/mRNA sequence between the 5’- and 3’-end RdRP-binding sites of samRNA, we can easily design and manufacture a variety of RNA/mRNA medicines and/or vaccines useful for developing all kinds of treatments against many human diseases, such as viral infections, cancers, diabetes, spinal muscular atrophy (SMA), Alzheimer’s disease, and many more.