Science and Technology
RNA MEDICINES THROUGH RNA EDITING
Most inherited rare diseases are caused by a single nucleotide variant in
the genome sequence. A splicing error, a misplaced termination, or an
erroneous transcription/translation can all corrupt the generated protein.
RNA editing may provide better therapeutic options that aim to fix
genetic problems by modifying and repairing the disease-causing RNA
transcript.
RNA editing includes the substitution of single bases, and/or the insertion
or deletion of larger nucleotide sequences. Within an RNA molecule,
substitution editing entails changing one nucleotide to another. The
hydrolytic deamination of adenosine to inosine is catalyzed by ADAR
enzymes. A-to-I editing can cause codon modifications since the
translational machinery reads inosine as guanosine. This means that
different amino acids are incorporated into the protein output to correct
the phenotype.
Unlike CRISPR-based therapies, RNA editing can target specific sites in an
RNA transcript without permanently changing a patient’s genome. RNA
editing is inherently safer to implement since it is reversible and does not
permanently alter the genetic code. We are thrilled about the RNA
editing space and the possibility for a new class of medications to
improve patients’ lives.
Our Proprietary
Platform
Our proprietary Lipid nanoparticle libraries and platforms are selectively targeted for successful delivery of RNA cargo to the target cells. LNPs act like tiny vehicles transporting their precious cargo “saRNA molecules” throughout the body. saRNA/LNP complexes travel through the bloodstream, dodging hazards and obstructions, until they arrive at their target. When they arrive, they unload their payload, allowing the molecules to start working.
Our proprietary nanoparticles have enhanced properties, such as particle stability, delivery efficacy, tolerability, biocompatibility, and biodistribution.
Accelerating Scientific Innovation in Self-amplifying RNA
Self-amplifying RNA vaccines are highly versatile. saRNA is beneficial compared to non-amplifying RNA as it has the advantages of rapid development, plug-and-play modular design, and cell-free synthesis, but with the added advantage of lower dose level due to self-replicating.
This reduces the burden of manufacturing, allowing facilities to make 100x more doses than with mRNA, lowering cost and time. Thus, in the case of a pandemic, rapid mass vaccines and distributed/decentralized manufacturing are feasible.
Therefore, preventing infectious diseases with our cutting-edge saRNA platform technology has limitless potential to meet unmet therapeutic needs.
Targeted Delivery
Mechanisms to Select Organs
An important and significant advantage to our platform is that saRNA will be formulated with novel and proprietary LNPs optimized for targeted delivery of saRNA-target to the lower respiratory tract and the potential to elicit protection against severe disease in the lungs.
Another advantage of our approach is the potential for nasal administration of saRNA/LNP. Nasal administration has the advantage of promoting strong mucosal immunity against circulating virulent, thus reducing upper respiratory tract infection and preventing transmission.
